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About Us
- Cure SMA’s 2019 Commitments to the Community
- Uniting the SMA Community Through Local Programs and Support
- Cure SMA Welcomes New Medical Advisory Council Committee Members
- Cure SMA Welcomes Aboard New Board and Committee Members
- Cure SMA Welcomes New Board and Committee Members
- Cure SMA Welcomes the Virginia Chapter
- Cure SMA Funds 26 Research Projects in 2014
- SMA Drug Pipeline Continues to Grow
- Applications for the Cure SMA Medical Advisory Board Due 10/1/14
- Funding Process Reaches Next Steps for Spinal Muscular Atrophy Clinical Care Research
- Cure SMA Announces New Research Funding
- Welcome to Cure SMA
Advocacy
- SMA Community Advocacy Wins and Champions Celebrated at Cure SMA Event
- Celebrating Disability Pride and the 34th Anniversary of the ADA at the White House
- Congressional Action Recognizes Unmet Needs of SMA Community
- SMA Community Advocacy Results in Key Accessible Air Travel Win
- HHS Action Improves Healthcare Accessibility and Strengthens Protections against Disability Discrimination in Healthcare
- Accessible Air Travel Update – Next Steps
- Government Funding Bill Supports New SMA Research to Address Unmet Needs
- Cure SMA Applauds DOT Proposal to Make Air Travel Safer for Wheelchair Users
- New Report Highlights Caregiving Challenges of Individuals with Spinal Muscular Atrophy
- 100% of States Now Screening Newborns for SMA
- Nevada Starts Screening for SMA – Only One State Remains!
- Administration Officials & Members of Congress Highlight SMA Community Advocacy at 12th Annual Hope on the Hill Washington, D.C. Event
- Cure SMA on Capitol Hill – A Message from Allie Williams
- Key Congressional Committee Urges NIH to Address Unmet Needs of Individuals with SMA
- New DOT Rule Will Require Truly Accessible Lavatories on New Single Aisle Aircraft
- Big Week in Washington for Cure SMA-Supported Legislation
- Congressional Committee Approves Legislation to Improve Air Travel for People with SMA and other Disabilities
- Cure SMA Applauds Federal Agency Decision that Should Lead to Better Insurance Coverage for Seat Elevation Systems in Power Wheelchairs
- Congress Examines Ways to Improve Air Travel for People with Disabilities
- District of Columbia Starts Screening for SMA During Rare Disease Month
- NEW REPORT: SMA Community Describes Air Travel Experiences as “Good, but mostly Bad, and Ugly”
- Cure SMA Unveils Advocacy 101 Video Series in Conjunction with 118th Congress
- Share Your Air Travel Concerns & Experiences for Cure SMA Report to Congress
- SMA Community Progress & Advocacy Featured at Cure SMA Hope on the Hill Event
- SMA Community Seeks SSI Improvements on Program’s 50th Anniversary
- Special NBS Awareness Month Announcement: South Carolina Screens for SMA!
- Special NBS Awareness Month Announcement: South Carolina Screens for SMA!
- Seat Elevators on Power Wheelchairs are a Necessity, not a Luxury, according to the SMA Community
- Cure SMA Advocacy Campaign Leads to Congressional Support for More SMA Research
- Cure SMA Announces Major Newborn Screening Milestone with Addition of Two New SMA Screening States
- In Just Three Years, Cure SMA Marks Nationwide Milestone for 85% of U.S. Babies Now Screened at Birth for SMA
- Community Spotlight: Xavier and Rose’s SMA Diagnosis
- Spring 2019 SMA Newborn Screening Update
- Cure SMA Advocates for SMA Community at ICER Hearing
- Community Spotlight: Kristen Smith
- Arkansas and Florida Adopt SMA Newborn Screening
- Register for Cure SMA’s Advocacy 101 Webinar: Learn How to Make a Difference
- Be A Part of Cure SMA’s Valentine’s Day Advocacy Campaign and Support the Newborn Screening Saves Lives Reauthorization Act
- Community Spotlight: Rebecca Smith and Micah Biello
- Hope on the Hill Congressional Dinner Raises $200,000
- Advocacy Year-in-Review 2018
- Closing out 2018 with Good News: Virginia Adopts SMA to their Newborn Screening Panel, Arizona Moves Closer to Adopting SMA Newborn Screening and Illinois Medicaid Covers Spinraza Treatment
- 8th Annual “Hope on the Hill” Congressional Dinner Will Be Held Later This Month in Washington D.C.
- Kansas Adopts SMA Newborn Screening as Several Other States Move Closer to Adoption
- North Carolina to Screen Newborns for SMA Through Early Check
- Community Spotlight: Adrienne Vollmer
- Community Spotlight: Allyson Henkel
- Community Spotlight: The Lasko Family
- Cure SMA’s Approach to Newborn Screening
- SMA Newborn Screening Advancements
- New York to Implement Newborn Screening for SMA October 1st
- Illinois Adopts Permanent SMA Screening
- Wisconsin Moves One Step Closer to SMA Newborn Screening
- HHS Secretary, Alex Azar, Recommends Nationwide Newborn Screening for SMA
- SMA Newborn Screening Moves Forward in New Jersey
- Ohio Moves One Step Closer to SMA Newborn Screening
- Indiana Becomes Fourth State to Adopt Newborn Screening for Spinal Muscular Atrophy
- SMA Advocates Meet With House and Senate Offices
- SMA Community Leaders to Gather in Washington DC to Network, Learn and Advocate
- Spring 2018 Issue of Compass Now Available Online
- House and Senate Vote to Increase Federal Funding for Newborn Screening Programs
- Indiana is the Fourth State to Adopt Permanent SMA Screening
- SMA Newborn Screening Moves Forward in Indiana, Ohio and Georgia
- #ShowYourRare and Raise SMA Awareness on World Rare Disease Day
- Advisory Committee on Heritable Disorders in Newborns and Children Recommends Nationwide Newborn Screening for Spinal Muscular Atrophy
- Utah Becomes First State to Implement Permanent Screening for SMA
- Federal Committee to Vote on Spinal Muscular Atrophy Newborn Screening on Thursday
- SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report
- Cure SMA Launches Advocacy Action Network
- Minnesota Becomes Second State to Adopt Newborn Screening for SMA
- Cure SMA Advocates Continue to Advance Newborn Screening Efforts
- SMA Advocates Testify at Federal Newborn Screening Meeting
- Cure SMA Meets in Utah with State and Federal Elected Officials to Support Newborn Screening
- Newborn Screening Advocacy Update
- SMA Families and Cure SMA Staff Visit Capitol Hill for Advocacy Efforts
- Missouri is the First State to Institute Newborn Screening for Spinal Muscular Atrophy
- Cure SMA Launches Newborn Screening Grassroots Advocacy Campaign for Spinal Muscular Atrophy Newborn Screening
- Call-to-Action: Senate Health Care Bill Released and Vote Expected Next Week
- Cure SMA Update – Senate Healthcare Bill
- SMA Newborn Screening Moves Forward in Missouri and Florida
- Cure SMA Advocacy Activities for the 2017 Year-to-Date
- Update: President’s Budget Proposes Significant Cuts to Federal Health Programs That Are Critically Important to SMA Patients and Families
- Anthem Updates Insurance Policy for Spinraza
- RUSP Nomination for SMA Accepted into Evidence Review
- Update on the Passage of the AHCA
- Cure SMA Grassroots Alert – AHCA
- Cure SMA Coverage and Payment Policy Project Continues to Move Forward
- Know Your Resources: How Congressional Caseworkers Can Help You Obtain Insurance Coverage for New Therapies
- SMA is Submitted to the Federal Newborn Recommended Uniform Screening Panel (RUSP)
- Cure SMA to Host Webinar with the FDA and Patient Advocates
- Registration Opens for the SMA Patient-Focused Drug Development Meeting with the FDA
- Updated SMA Drug Pipeline Released as First Commercial Patient Receives Spinraza
- SMA Patient-Focused Drug Development Meeting with FDA Set for April 18, 2017
- Join Us for an Upcoming Webcast about SPINRAZA TM (nusinersen)
- FDA Approves Spinraza for SMA
- Cure SMA Participates in a Special FDA Listening Session
- SMA Community Conducts Outreach Visits with Members of Congress
- Annual Congressional Dinner Brings SMA Community Together on the Eve of 21st Century Cure Bill Passage
- Help Us Shape the Future Together, and Reach our Goal of $750,000
- Cure SMA Launches Insurance Coverage and Payment Policy Project
- Cure SMA Joins SMA Europe and TREAT-NMD for Meeting with EMA Regulators
- Cure SMA to Host Webinar on the New Drug Application Process
- 6th Annual Congressional Dinner Furthers SMA Advocacy Work
- SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA
- Cure SMA Announces Newborn Screening Initiative in Partnership with MDA
- We’re Partnering With The Mighty!
- Cure SMA Plans a Series of Educational Modules for the SMA Community
- Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy
- Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports
- Cure SMA Posts Keynote Presentations on the NDA Process
- 2016 Update on SMA-FDA Interactions
- Cure SMA and the SMA Community Featured by Astellas Pharmaceuticals
- Global Genes Features Cure SMA in Patient Voice Project
- Senate HELP Committee Passes Another 7 Innovation Bills
- Senate HELP Committee Passes Innovation Bills
- Update on 21st Century Cures/Innovation: the Senate’s “Step-by-Step” Approach
- Cure SMA Testifies Before FDA at PDUFA Meeting
- 5th Annual Congressional Dinner Brings Together Families, Government and Industry
- 5th Annual Hope on the Hill Congressional Dinner Advances SMA Advocacy
- Clinical and Regulatory Webinar and Extended Q&A Now Available
- Tell Your Senator to Support Innovations for Healthier Americans
- Cure SMA Publishes Journal Article on the Patient Voice
- House of Representatives Passes Ensuring Access to Clinical Trials Act
- FDA Engagement Update: The Voices of SMA
- House of Representatives Passes 21st Century Cures
- Cure SMA Announces Partnership with The District Policy Group
- Cure SMA Receives NIH Grant
- 21st Century Cures Unanimously Passes Committee
- Cure SMA Joins The Research Acceleration and Innovation Network
- Update on SMA-FDA Interactions
- New 21st Century Cures Discussion Draft Released
- 21st Century Cures Discussion Document Released
- Hope on the Hill Advances Key SMA Legislative Priorities
- Families, Government, and Industry Leaders Unite to Help Cure SMA
- Urge Your Senators to Support Passage of the Newborn Screening Saves Lives Reauthorization Act
- Call Congress and Urge Passage of The ABLE Act
Archives
- Cure SMA Awards $150,000 to Dr. Lee Rubin at Harvard to Advance a New SMA Drug Discovery Program
- Pfizer Licenses Cure SMA Quinazoline Drug Program from Repligen
- Cure SMA Announces Grant to Cytokinetics for Preclinical Development of Tirasemtiv for Spinal Muscular Atrophy
- ISIS Pharmaceuticals Initiates Clinical Study in Infants with Spinal Muscular Atrophy
- Cure SMA Awards $150,000 to Dr. Burghes and Dr. Lorson to Investigate New Antisense Therapies for Spinal Muscular Atrophy
- VALIANT Clinical Trial of Ambulatory Type III Spinal Muscular Atrophy Patients Conducted at Ohio State University Published in Muscle and Nerve
- Cure SMA Awards $150,000 to Drs. Hua and Krainer at CSHL to Investigate Antisense Oligonucleotides for Spinal Muscular Atrophy
- PTC Therapeutics Announces Selection of a Drug Development Candidate for Spinal Muscular Atrophy
- Meet Newly Funded Cure SMA Researcher: Dr. Adrian Krainer
- Meet Newly Funded Cure SMA Researcher: Dr. Yimin Hua
- Cure SMA Scientific Advisory Board Highlights Findings from The 2013 SMA Research Group Meeting
- Meet Newly Funded Cure SMA Researchers: Oscar Mayer, MD PhD and Vanessa Battista, MS
- Meet Newly Funded Cure SMA Researcher: Dr. Kathryn Swoboda
- Meet Newly Funded Cure SMA Researcher Dr. Timothy Lotze
- Meet Newly Funded Cure SMA Researcher Dr. Matthew A. Halanski
- Cure SMA Contributes Research Funding to Sixteen SMA Journal Articles in 2013
- Cure SMA Funds 25 Research Projects in 2013
- Trophos Completes Efficacy Study of Olesoxime in Patients with Spinal Muscular Atrophy
- Isis Reports Follow-Up Data From Phase 1 Study In Children With Spinal Muscular Atrophy
- Pfizer RG3039 Program for Spinal Muscular Atrophy Reaches Development Milestone
- PTC Therapeutics and Roche Announce Spinal Muscular Atrophy Drug Enters First Stage of Clinical Development
- Cure Spinal Muscular Atrophy Awards $600,000 for Basic Research to Identify New Approaches for SMA Therapies
- Cure Spinal Muscular Atrophy Awards New Research Funding of $90,000 to Drs. Bogdanik and Lutz at The Jackson Laboratory
- Cure Spinal Muscular Atrophy Awards New Research Funding of $95,000 to Dr. Barrington Burnett
- 100 Research Presentations at The 2014 Spinal Muscular Atrophy Research Group Meeting Hosted and Supported by Cure SMA
- Cure Spinal Muscular Atrophy Awards New Research Funding of $95,000 to Dr. Allison Ebert
- Cure Spinal Muscular Atrophy Awards New Research Funding of $95,000 to Dr. Kejun Han
- Cure Spinal Muscular Atrophy Awards New Research Funding of $100,000 to Dr. Rashmi Kothary
- Cure Spinal Muscular Atrophy Awards New Research Funding of $75,000 to Dr. Chien-Ping Ko
- Regenx Biosciences and Avexis Enter into Agreement for The Development of Treatments for Spinal Muscular Atrophy Using NavRaav9 Vectors
- Cure Spinal Muscular Atrophy is Currently Funding 24 Research Projects with Multiple Rounds of New Funding to be Announced in 2014
- Cure SMA Awarded Grant from the National Institute of Health for the 2014 Spinal Muscular Atrophy Research Group Meeting
- Cure SMA Releases a Request for Proposals for New Funding of Research Projects for Spinal Muscular Atrophy.
- Cure SMA Releases a New Request for Proposals for Clinical Care Research Projects in Spinal Muscular Atrophy
- Update from Dr. Lorson at University of Missouri on Spinal Muscular Atrophy Program being Funded by Cure SMA
- Cure SMA Announces Basic Research Funding for Dr. Lyndsay Murray
- Cure SMA Honors Scott Geller with 1st Annual Community Partnership Award
- Fundraising Support of Cure SMA Reaches New Milestone
- Start a Fundraiser with These Resources and Ideas from Cure SMA!
- BAYADA Pediatrics Renews National Sponsorship with Cure SMA
- Spring Milestone Events Reaching New Heights for Cure SMA
- Join Cure SMA at the Ballparks to Raise Awareness for Spinal Muscular Atrophy
- Record Breaking Fundraising Year in Support of Cure Spinal Muscular Atrophy
- Meet Your Fundraising Goals with These Resources and Tips from Cure SMA
- SMA Community Mourns the Passing of David Cunniff
- SMA Families Make The Annual SMA Conference Possible
- BAYADA Pediatrics Renews $25,000 Sponsorship of the Cure SMA Walk-n-Roll Program
- SMA Families Strength Awareness of Spinal Muscular Atrophy Throughout the Month of August
- SMA Awareness Month: What You Can Do with Support From Cure SMA!
- Cure SMA Welcomes the Georgia Chapter
- Cure SMA Welcomes the New Carolinas Chapter and Satellite Chapter Capital Region of Greater New York
- Another Record Breaking Fall Fundraising Season in Support of Cure SMA
- SMA Families from Across the Country Make Waves During SMA Awareness Month
- SMA Awareness Month: What You Can Do with Support From Cure SMA!
- Cure SMA Announces #GivingTuesday Social Media Campaign
- Cure SMA Announces #GivingTuesday Social Media Campaign
- SMA Researchers Talk About the Importance of the Spinal Muscular Atrophy Research Group Meeting
- Cure SMA Releases Keynote Speeches and Drug Companies Summaries from The 2014 Annual Spinal Muscular Atrophy Conference
- Trophos Presents Pivotal Phase II/III Study Results on Olesoxime in Spinal Muscular Atrophy at The American Academy of Neurology Meeting
- Isis Presents Update on Spinal Muscular Atrophy Drug Trials at the American Academy of Neurology Meeting
- AveXis Announces Dosing of First Patient for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type I
- Tirasemtiv Demonstrates Improvements in Muscle Force, Grip Strength and Resistance to Fatigue in Preclinical Mouse Models of Spinal Muscular Atrophy
Clinical Trials
- New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA
- Novartis Gene Therapies Releases SMA Community Statement
- Biogen Shares DEVOTE and RESPOND Study Updates
- NMD Pharma Initiates Phase II Trial for SMA
- Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial
- Biohaven Completes Enrollment in Pivotal Phase 3 Study
- Clinical Research and Drug Development Session Highlights from 2023 SMA Research & Clinical Care Meeting
- Cure SMA Releases Updated Drug Pipeline
- Update on Genentech/Roche Initiation of MANATEE Clinical Study
- Novartis Announces Lift of Partial Clinical Trial Hold, Plans to Initiate a New Study of Intrathecal Zolgensma in Older Patients with SMA
- Data for Genentech’s Evrysdi Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies with Type 1 SMA
- Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children
- Community Statement from Novartis Gene Therapies
- Genentech Shares New Evrysdi Data on Improved Motor Function and Survival in Babies with SMA Type 1
- Biogen Shares New Spinraza Data Exploring Opportunities to Improve Outcomes for SMA Patients
- Scholar Rock Community Statement on TOPAZ 12-Month Topline Data
- Nusinersen Research Updates Presented by Biogen
- Research Updates from Scholar Rock
- Evrysdi Research Updates from Genentech
- Zolgensma Research Updates from Novartis Gene Therapies
- Biogen SMA Q1 2021 Community Statement
- Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM
- Cure SMA Offer Perspectives on Combination Therapy for SMA
- Biogen Provides SMA Community Update on DEVOTE Study
- Scholar Rock Issues Community Statement with Update on SRK-015 in Patients with Types 2 and 3 SMA
- Genentech Presents New 2-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA
- Novartis Issues Community Statement with Update on AVXS-101 Intrathecal Clinical Development Program
- Biogen Plans to Initiate Phase 4 Study Evaluating Benefit of Spinraza in Patients Treated with Zolgensma
- Cure SMA to Fund Network of Prominent SMA Clinical Research and Treatment Centers
- Cure SMA Publishes Article on SMA Clinical Trial Readiness Program in the U.S.
- Biogen Shares SPINRAZA Research Update
- Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 Years with Type 2 or Type 3 SMA
- Genentech’s Risdiplam Meets Primary Endpoint in Pivotal FIREFISH Trial in Infants with Type 1 SMA
- Community Statement from Scholar Rock Regarding SRK-015
- Cure SMA and Biogen to Co-Host Webinar on DEVOTE Clinical Trial – Register Today!
- Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study, DEVOTE, Evaluating a Higher Dose of SPINRAZA and Additional Data in a Broad Range of Patients
- Biogen Issues Community Statement on Spinraza
- Biogen Announces New NURTURE Data at the 2019 Annual SMA Conference
- Cytokinetics Releases New Data from Reldesemtiv Clinical Study at the 2019 Annual SMA Conference
- Biogen Issues Q2 Community Statement on Spinraza
- Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers
- AveXis Issues Community Statement on Zolgensma Approval
- AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age
- Novartis Issues Community Statement on Branaplam Clinical Study
- Scholar Rock Issues Community Statement on the Initiation of a New Clinical Trial for SRK-015
- Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in Spinal Muscular Atrophy at the AAN Annual Meeting
- AveXis Presents Data at AAN Annual Meeting Demonstrating Efficacy of Zolgensma in Broad Spectrum of Spinal Muscular Atrophy (SMA) Patients
- Biogen to Present Data at AAN Highlighting Spinraza and Emerging Biomarkers for SMA
- Genentech Presents New Data at the 2019 AAN Annual Meeting
- Biogen Presents New Data Regarding Adults and Infants with SMA
- The Winter 2019 issue of Compass in Now Available Online
- Recording of Genentech/Roche and Cure SMA Webinar Now Available Online
- Biogen Provides 2019 Community Update on Spinraza
- Genentech/Roche and Cure SMA to Co-Host Webinar
- Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans
- Cytokinetics Announces Receipt of FDA Feedback Regarding Reldesemtiv in Patients with SMA
- Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam
- December Webinar on SMA Treatments and Clinical Trials Now Available Online
- Biogen Issues Q4 Community Statement on Spinraza
- AveXis Issues Community Statement on BLA Acceptance
- Novartis Announces FDA Filing Acceptance and Priority Review for AVXS-101
- Register for Cure SMA’s Webinar on SMA Treatments and Clinical Trials
- Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of SMA
- AveXis Issues Community Statement on FDA Filing for SMA Type I
- AveXis Files for FDA Approval of Gene Therapy for Spinal Muscular Atrophy
Type I - New Spinraza Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with SMA
- Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA
- Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress
- Genentech Releases Community Statement on Risdiplam
- Roche Releases Community Update on Risdiplam
- PTC Therapeutics Announces Risdiplam (RG7916) is Well Tolerated at All Dose Levels With No Drug-Related Safety Findings
- Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference
- Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015
- Roche Releases Community Statement on Olesoxime Program
- Biogen Releases Community Statement on Spinraza Access and New Data
- AveXis to Present Phase 1 Data at the 70th American Academy of Neurology Annual Meeting
- Biogen Presents New Data at the American Academy of Neurology (AAN) Annual Meeting
- Genentech to Present New RG7916 Data at the American Academy of Neurology (AAN) Annual Meeting
- Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with SMA
- Roche Releases Community Statement on RG7916 and Olesoxime Programs
- Biogen Announces New Interim Phase 2 Results From NURTURE
- Phase 2 Clinical Trial Results for CK-2127107 Expected in Second Quarter of 2018
- Biogen Releases Statement on CHERISH Data and Plans for New Clinical Research
- Preliminary Data from FIREFISH Trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy
- AveXis Releases Community Statement on Expanded Clinical Trials
- Cure SMA to Host Webinar on Treatment Access and Clinical Trials on February 15
- 2018 Updated SMA Drug Pipeline Released
- AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy
- Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials
- AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
- Cytokinetics Publishes Clinical Trial Data for CK-2127107
- Biogen Issues Community Update on Spinraza
- New Clinical Trial Data Published in New England Journal of Medicine
- Genentech/Roche Releases Clinical Trial Update for SUNFISH (RG7916)
- AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process
- Novartis Releases Update on LMI070 (Branaplam) Clinical Trial
- Genentech/Roche Releases Clinical Trial Update for RG7916
- Roche Opens US Recruitment for Phase 2 Clinical Trial
- Cytokinetics Receives FDA Orphan Drug Designation for CK-2127107 for SMA
- Biogen Releases New Data from Cherish and Nurture Trials
- Cytokinetics Announces Start of Second Cohort in Phase 2 Clinical Trial of CK-2127107 in Patients with SMA
- Genentech Roche Releases Community Letter with Clinical Trial Updates
- AveXis Reports Results from Phase 1 Trial of AVXS-101
- Biogen Presents New ENDEAR Data Showing SPINRAZA (nusinersen) Significantly Reduces Risk of Death or Permanent Ventilation
- Join Us for an Upcoming Webcast about SPINRAZA TM (nusinersen)
- Biogen Provides Community Statement and Q&A on the Approval of Spinraza for SMA
- Ionis Pharmaceuticals Issues Community Statement on the Approval of Spinraza
- FDA Approves Spinraza for SMA
- The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants
- Biogen and Ionis Community Statement on Interim Analysis of CHERISH (Type 2) Trial
- Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study
- AveXis Announces Single-Arm Design for Pivotal Study of AVXS-101
- AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 at the World Muscle Society Congress
- Biogen Presents New Data at World Muscle Society Congress
- Biogen and Ionis Release Community Statement on NDA Filing Completion
- Roche Launches New Phase 2 Clinical Trials
- Roche Provides New Update on Clinical Development of RG7916
- 2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function
- 2016 SMA Researcher Meeting Summary: SMA Therapy Development
- 2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 2
- 2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 1
- Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene
- 2016 SMA Researcher Meeting Summary: Clinical Research Studies
- Cure SMA Plans a Series of Educational Modules for the SMA Community
- AveXis Reports Second Quarter 2016 Interim Data from Ongoing Phase 1 Trial of AVXS-101 (Gene Therapy)
- 2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA
- Novartis Releases Community Update on the Study of LMI070
- Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings
- Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA
- AveXis Receives FDA Breakthrough Therapy Designation for AVXS-101 (Gene Therapy) for SMA
- Cure SMA Releases 2016 Updates from Drug Programs in Clinical Trials
- Cure SMA Releases June 2016 Update to the SMA Drug Pipeline
- Biogen and Ionis Release Spring 2016 Community Update
- Update on the Clinical Development of LMI070
- AveXis Reports Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1
- Ionis Releases Data Update from Phase 2 Study in Infants with SMA
- Annual SMA Conference to Host Panels on NDA Process and SMA Drugs in Development
- AveXis Releases New Data on Phase 1 Clinical Trial
- Cure SMA-Funded Researcher Publishes Paper on Biomarker Study
- Biogen and Ionis Provide Update on Nusinersen (IONIS-SMNRx)
- Roche Announces New Clinical Trial of RG7916
- AveXis Announces the Completion of Enrollment for Phase 1 Gene Therapy Trial
- Cytokinetics Opens Enrollment for Phase 2 Trial
- Cure SMA Funds 28 Research Projects in 2015, as Drug Pipeline Grows to 18 Programs
- Clinical and Regulatory Webinar and Extended Q&A Now Available
- Cure SMA and Other Venture Philanthropy Leaders Share Insight at Partnering for Cures Panel
- Cytokinetics Announces New Details on Upcoming Phase 2 Trial
- Isis Pharmaceuticals Announces SHINE, an Open-Label Extension Study
- Cure SMA to Host Clinical and Regulatory Webinar
- PTC Announces New Clinical Trial Results at World Muscle Congress
- Community Update on ISIS-SMNRx from Biogen and Isis
- New Gene Therapy Results Announced at World Muscle Congress
- New Update on Clinical Development of RG7800
- 2015 SMA Researcher Meeting Summary: Clinical Research
- Updates from Drug Programs in Clinical Trials
- Cytokinetics Releases Update on Phase 1 Trial at Annual SMA Conference
- Isis Announces Additional Trial Results at Annual SMA Conference
- Cure SMA Announces Nearly $2 Million in New Planned Research Funding
- Read the Summer 2015 Issue of Compass
- Cure SMA Releases New Clinical Trial Care Series Booklet
- Isis Releases Encouraging Results from Phase 2 Study
- SMA Drug Pipeline Grows to 18 Programs
- Biogen Announces EMBRACE, a New Phase 2 Clinical Trial
- Novartis Launches Phase 2 Clinical Trial
- Cytokinetics and Astellas Release Further Details on Upcoming Phase 2 Trial
- Update on Clinical Development of RG7800
- Annual SMA Conference to Feature Panel of Leading SMA Drug Experts
- Biogen Announces NURTURE, a New Phase 2 Clinical Trial
- Updates on Pharmaceutical Companies Investing in SMA Drug Development
- Roche Buys Trophos to Continue Work on SMA Drug
- NIH Releases Summary of the SO-SMART Workshop
- Cytokinetics and Astellas Announce New Phase 2 Clinical Trial
- Isis Expands Phase 3 Clinical Trials
- SMA Drug Pipeline Continues to Grow
- PTC Therapeutics Announces New Phase 1a/2b Clinical Trial
- Isis Announces Second Phase III Clinical Trial
- 2014 SMA Researcher Meeting Summary: Clinical Trials
- 2014 SMA Researcher Meeting Summary: Therapy Development
- Isis Releases Phase II Clinical Trial Results
- AveXis Announces the Completion of Dosing of the Low-Dose Cohort in US Clinical Trial for Spinal Muscular Atrophy
- Isis Presents Phase II Trial Results
- AveXis Receives Orphan Drug Designation
- Isis Begins Recruiting for Phase III Trial
- Spinal Muscular Atrophy Biomarker Study Reaches Enrollment Goals
- Cure SMA and PPMD Invite Spinal Muscular Atrophy Parents to Take Part in Two Clinical Trial Surveys
- Spinal Muscular Atrophy Mice Model Results Published in the Journal Science
- Isis Begins Phase III Clinical Trial
Community Awareness
- August is Spinal Muscular Atrophy (SMA) Awareness Month!
- SMA Community Advocacy Results in Key Accessible Air Travel Win
- Celebrate Rare Disease Day with Cure SMA
- Cure SMA Applauds DOT Proposal to Make Air Travel Safer for Wheelchair Users
- Cure SMA is Thrilled to Announce the Establishment of our New Chapter in Puerto Rico!
- August is SMA Awareness Month!
- Cure SMA Holds Patient-Led Listening Session with FDA
- Cure SMA Holds Patient-Led Listening Session with FDA
- Community Spotlight: Shaakira Thomas and Family
- Community Spotlight: Dr. Arthur Burghes
- Community Spotlight: Brian Chiorello
- Community Statement from AveXis Regarding Commitment to SMA Community Amid COVID-19 Pandemic
- Community Statement from Genentech on Risdiplam Approval Timeline
- Celebrate Rare Disease Day 2020 with Cure SMA
- Lights…Camera…Action! Search Is On for Child with SMA to Appear in NBC Television Pilot
- Community Statement from Scholar Rock Regarding SRK-015
- Community Spotlight: Angela Wrigglesworth
- Community Spotlight: Steven Verdile
- Cure SMA and Ionis Announce Holiday Card Contest
- Cure SMA Partners with the Clements Twins and Nail & Bone
- SMA Awareness Month 2019 Has Begun!
- Community Spotlight: Laura Watson
- Team Cure SMA to Participate in Vermont City Marathon
- Community Spotlight: Kyle and Laura Derkowski
- Community Update Survey Q&A
- What is the Summit of Strength Program?
- Team Cure SMA New York City Half Marathon Runners Raise $15,000 for Cure SMA
- Cure SMA Launches Third Annual Community Survey to Address Important Issues in SMA Treatment
- New Dates and Locations Announced for 2019 Summits of Strength
- Community Spotlight: Kristen Smith
- Arkansas and Florida Adopt SMA Newborn Screening
- Community Spotlight: Amber Bosselman
- Show Your Rare. Show You Care.
- Register for Cure SMA’s Advocacy 101 Webinar: Learn How to Make a Difference
- The Cure SMA 2019 Goal List
- Cure SMA’s 2019 Commitments to the Community
- Cure SMA is Thrilled to Launch Its New and Refreshed Endurance Program: Team Cure SMA
- Be A Part of Cure SMA’s Valentine’s Day Advocacy Campaign and Support the Newborn Screening Saves Lives Reauthorization Act
- Heather Tomko on Goal-Setting for the New Year
- Fall 2018 Issue of Directions Now Available
- Congratulations to the Winners of Cure SMA’s Video Contest!
- Community Spotlight: Rebecca Smith and Micah Biello
- Community Spotlight: The Jankowski Family
- Community Spotlight: The Berkovits Family
- Closing out 2018 with Good News: Virginia Adopts SMA to their Newborn Screening Panel, Arizona Moves Closer to Adopting SMA Newborn Screening and Illinois Medicaid Covers Spinraza Treatment
- Cure SMA Launches “SMArt Moves” for Early Diagnosis and Treatment
- Cure SMA is Thrilled to Launch a New Support Program for Teens and Adults with SMA
- Summit of Strength Program Receives Positive Feedback and Increases Attendance
- Meet the NDEAM Webinar Panelists
- Meet the National Disability Employment Awareness Month (NDEAM) Panelists
- NDEAM Spotlight: Jaclyn Greenwood
- National Disability Employment Awareness Month Spotlight: Kim Hill
- Register for Cure SMA’s First National Disability Employment Awareness Month Webinar
- Cure SMA Receives Generous Funding from Luke 18:1 Foundation
- SMA Care Center Network and Clinical Data Registry Launched
- Community Spotlight: Adrienne Vollmer
- Cure SMA Launches a Family Support App for Your Mobile Device!
- Community Spotlight: Allyson Henkel
- Community Spotlight: The Lasko Family
- Sweet Baby Zane Funds New Equipment Pool Item
- Cure SMA Receives Generous Donation from the Cash Scanlon-Philips Foundation
- Community Spotlight: The Anton Jensen Family
- Community Spotlight: The Nelson Family
- Community Spotlight: The Zmaczynski Family
- Community Spotlight: Doug McCullough
- Cure SMA Concert for a Cure Reaches $2 Million Milestone
- Community Spotlight: The Colone Family
- Heather Tomko Crowned as Ms. Wheelchair USA 2018
- Community Spotlight: Brynne Willis
- August is SMA Awareness Month!
- Thank You For a Wonderful 2018 Annual SMA Conference!
- Traveling with SMA
- SMA Community Leaders to Gather in Washington DC to Network, Learn and Advocate
- SMA Newborn Screening Guidelines Published in Journal of Neuromuscular Diseases
- Community Spotlight: Nathan Yates
- The Discovery of SMA
- Cure SMA Launches Second Annual Community Survey to Address Important Issues in SMA Treatment
- Indiana is the Fourth State to Adopt Permanent SMA Screening
- Awareness is Best Raised When We Work Together
- SMA Newborn Screening Moves Forward in Indiana, Ohio and Georgia
- #ShowYourRare and Raise SMA Awareness on World Rare Disease Day
- Last Week’s Webinar on Treatment Access and Clinical Trials Now Available
- Cure SMA Receives Generous Gift from the Weisman Family Foundations
- Cure SMA Receives Generous Gift
- Cure SMA Announces Local Educational Symposiums in 16 US Locations
- Cure SMA to Host Webinar on Treatment Access and Clinical Trials on February 15
- Miller McNeil Woodruff Foundation Awards Cure SMA Generous Gift
- Fall 2017 Directions Now Available for Download
- Cure SMA Advocates Continue to Advance Newborn Screening Efforts
- 2017 World Muscle Society Congress Recap
- Cure SMA Launches Benefit-Risk Survey to Address Important Issues in Treatment Access and Future Drug Approvals in SMA
- Recording of the September Webinar on Spinraza Updates Now Available
- Newborn Screening Advocacy Update
- 2017 SMA Awareness Month Recap
- Cure SMA to Host Webinar Updating the Community on Spinraza Access
- SMA Documentary Premieres at 2017 Rhode Island International Film Festival
- August is SMA Awareness Month!
- SMA Newborn Screening Moves Forward in Missouri and Florida
- Cure SMA Advocacy Activities for the 2017 Year-to-Date
- SMA Community’s Voice Heard “Loud and Clear” at Last Week’s Patient Focused Drug Development Meeting with the FDA
- Cure SMA to Host Webinar on Spinraza Access, as List of Administration Sites is Released
- Cure SMA Publishes Second Journal Article on the Patient Voice
- Know Your Resources: How Congressional Caseworkers Can Help You Obtain Insurance Coverage for New Therapies
- Cure SMA to Host Webinar with the FDA and Patient Advocates
- Spinraza Access Update Webinar
- Cure SMA Launches Community Survey to Address Urgent and Important Issues in SMA Treatment
- Updated SMA Drug Pipeline Released as First Commercial Patient Receives Spinraza
- SMA Patient-Focused Drug Development Meeting with FDA Set for April 18, 2017
- Join Us for an Upcoming Webcast about SPINRAZA TM (nusinersen)
- Biogen Provides Community Statement and Q&A on the Approval of Spinraza for SMA
- Ionis Pharmaceuticals Issues Community Statement on the Approval of Spinraza
- Cure SMA Participates in a Special FDA Listening Session
- The Miller McNeil Woodruff Foundation Makes a Generous Contribution to Cure SMA
- Annual Congressional Dinner Brings SMA Community Together on the Eve of 21st Century Cure Bill Passage
- Annual South Florida Gala of Hope Raises over $1 Million in 6 Years
- Cure SMA Joins SMA Europe and TREAT-NMD for Meeting with EMA Regulators
- Cure SMA to Host Webinar on the New Drug Application Process
- SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA
- Cure SMA and Ionis Announce 2016 Holiday Card Contest
- Cure SMA Announces Newborn Screening Initiative in Partnership with MDA
- Cure SMA Welcomes Aboard New Board and Committee Members
- We’re Partnering With The Mighty!
- Cure SMA Plans a Series of Educational Modules for the SMA Community
- Cure SMA Releases Full Schedule for the 2016 Annual SMA Conference
- Cure SMA Welcomes New Board and Committee Members
- Cure SMA and the SMA Community Featured by Astellas Pharmaceuticals
- Adult Scholarships Offered for 2016 Annual SMA Conference
- Global Genes Features Cure SMA in Patient Voice Project
- Winter 2015 Issue of Directions Now Online
- Patients and Caregivers of Patients with Type II and III SMA Needed for Educational Research Survey
- Cure SMA Receives Generous Donation from Fighting for Kaiden Foundation
- 5th Annual Congressional Dinner Brings Together Families, Government and Industry
- Cure SMA and Cytokinetics Announce Partnership to Advance Education and Awareness of Spinal Muscular Atrophy
- 5th Annual Hope on the Hill Congressional Dinner Advances SMA Advocacy
- Cure SMA Welcomes the North Jersey Chapter
- Cure SMA and Other Venture Philanthropy Leaders Share Insight at Partnering for Cures Panel
- Cure SMA to Host Clinical and Regulatory Webinar
- Thank You to Jessica Moyer and Family
- FDA Engagement Update: The Voices of SMA
- Cure SMA and Isis Announce 2015 Holiday Card Contest
- Western New York Teen Wins Nickelodeon Halo Award
- SMA Awareness Month Breakthroughs
- Seven Individuals and Families Recognized for Their Contributions to the Community
- Spring 2015 Directions Posted Online
- In Memory of Ken Emerson
- Meet the SMA Family that Helped Inspire 21st Century Cures
- Cure SMA Joins The Research Acceleration and Innovation Network
- Update on SMA-FDA Interactions
- New 21st Century Cures Discussion Draft Released
- Share Your SMA Story for a New Research Project
- Cure SMA Welcomes the Virginia Chapter
- Cure SMA Releases Updated Overview of Research Model
- How Our Cure SMA Community is Raising Awareness
- Cure SMA Annual Campaign Sets $1 Million Goal
- 21st Century Cures Discussion Document Released
- Join Cure SMA in Celebrating the International Day of Acceptance
- Together, We Made History
- NIH Releases Summary of the SO-SMART Workshop
- Fall 2014 Issue of Directions Now Online
- Erin Trainor Memorial Fund Reaches Milestone
- Miller McNeil Woodruff Foundation Gives Generous Gift to Cure SMA
- Hope on the Hill Advances Key SMA Legislative Priorities
- Families, Government, and Industry Leaders Unite to Help Cure SMA
- Together, We Can Cure SMA
- BAYADA Pediatrics Renews National Sponsorship with Cure SMA
- Cure SMA Honors Scott Geller with 1st Annual Community Partnership Award
- Join Us in Kansas City
- Cure SMA is Groupon’s Charity of the Day
- Cure SMA Announces New Subcommittee on Adults with SMA
- Cure SMA Receives Generous Donation from Barney & Barney
- Cure SMA and Isis Announce Holiday Card Contest TEST
- Cure SMA and Isis Announce Holiday Card Contest
- Cure SMA and PPMD Invite Spinal Muscular Atrophy Parents to Take Part in Two Clinical Trial Surveys
- Chef Takashi Competes for Cure SMA
Community Spotlight
- Community Spotlight: Viola Dwyer, SMA Community Advocate from Pennsylvania
- A Community’s Unwavering Love: How the Stickane Family Started a Foundation to Cure Spinal Muscular Atrophy
- Community Spotlight: Holly Ianno and Family
- Community Spotlight: Dee and River Rolle
- Community Spotlight: Sarah Manuel
- Donor Spotlight: Nieves and Valerie Gomez
- Community Spotlight: Paula Lavigne and Chris Arnold
- Community Spotlight: Michelle Tynski and Family
- Community Spotlight: Amber-Joi Watkins and Family
- Community Spotlight: Alexandra and Joe Lakhman
- Community Spotlight: Shaakira Thomas and Family
- Community Spotlight: Dr. Arthur Burghes
- Community Spotlight: Doug McCullough
- Community Spotlight: Chan Family
- Community Spotlight: Elizabeth Lockwood
- Community Spotlight: Nick Farrell
- Community Spotlight: I am…Viola Dwyer
- Jessica’s Story: Remembering Piper Grace
- Community Spotlight: I Am…Kelsey Roberts
- Community Spotlight: Brian Chiorello
- Community Spotlight: The Hoskin Family
- Community Spotlight: Nikki McIntosh
- Community Spotlight: Sarah Boggess, Research Coordinator, Ornamental Plant Pathology
- Community Spotlight: Perspectives from Spencer Cook, Registered Accessibility Specialist
- Community Spotlight: Kayley Shade of Sioux Falls Storm
- Community Spotlight: AJ Brockman of Brewhouse Gallery and Kelsey City Brewing Company
- Community Spotlight: Xavier and Rose’s SMA Diagnosis
- Community Spotlight: Braedon’s Newborn Screening Diagnosis
- Community Spotlight: Nicole Stickane Advocates for Newborn Screening in Texas
- Community Spotlight: Avery’s Diagnosis from Newborn Screening
- Community Spotlight: Ben Lou’s Formula for Life
- Community Spotlight: Raising Money for Cure SMA, One Cookie at a Time
- Community Spotlight: Cesar Rodrigues
- Community Spotlight: The Forcier Family
- Community Spotlight: Scurria Family
- Community Spotlight: The Veit Family
- Community Spotlight: Maria Kaloidis
- Community Spotlight: David Alvarez
- Caring for the Caregiver by Brynne Willis
- A Tiny Warrior: Winnie Stanley’s Story
- Community Spotlight: Angela Wrigglesworth
- Community Spotlight: Steven Verdile
- Community Spotlight: Paul Guarino of PG Sports
- Community Spotlight: Laura Watson
- Community Spotlight: Kyle and Laura Derkowski
- Community Spotlight: Kristen Smith
- Community Spotlight: Amber Bosselman
- Heather Tomko on Goal-Setting for the New Year
- Community Spotlight: Rebecca Smith and Micah Biello
- Community Spotlight: The Jankowski Family
- Community Spotlight: The Berkovits Family
- Community Spotlight: Adrienne Vollmer
- Community Spotlight: Allyson Henkel
- Community Spotlight: The Lasko Family
- Community Spotlight: The Anton Jensen Family
- Community Spotlight: The Nelson Family
- Community Spotlight: The Zmaczynski Family
- Community Spotlight: Doug McCullough
- Community Spotlight: The Colone Family
- Community Spotlight: Brynne Willis
- Community Spotlight: Nathan Yates
Conference
- Thank You for an Amazing 2024 Annual SMA Conference!
- SMA Scientists and Clinicians from Around the World Gather at the 27th Annual SMA Research & Clinical Care Meeting
- Thank You for an Amazing 2023 Annual SMA Conference!
- Individuals with SMA Describe New Travel Hiccups and Wheelchair Damage in Their Air Travels to Cure SMA National Conference
- Luke 18:1 Foundation Donates $500,000 to Cure SMA to Support Groundbreaking SMA Research and Care
- 2022 Cure SMA Annual Research Meeting Summaries – Special and Clinical Research Session
- 2022 Cure SMA Annual Research Meeting Summaries – Basic Research Sessions
- Thank You for a Wonderful 2022 Annual SMA Conference!
- Thank You for Another Fantastic Virtual SMA Conference!
- Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA
- New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA
- Community Spotlight: Dr. Arthur Burghes
- Understanding the Benefits of Aqua Therapy
- Ready to Go! Platform Open for 2021 Virtual SMA Conference Events
- Cure SMA Announces 2022 Annual SMA Conference
- Thank You for a Wonderful 2020 Virtual SMA Conference!
- Genentech Announces 2-Year Risdiplam Data From SUNFISH and New Data From JEWELFISH in Infants, Children and Adults with SMA
- Biogen Shares Results from Landmark NURTURE Study of Pre-Symptomatic SMA Patients Treated with Spinraza
- 2020 Virtual SMA Conference Booklet is Now Available Online
- Registration Now Open for the 2020 Virtual SMA Conference
- Cure SMA Announces a 2020 Virtual SMA Conference – United, At Home
- Cure SMA Announces Cancellation of 2020 Annual SMA Conference and Research & Clinical Care Meeting
- 2020 Annual SMA Conference Schedule is Released
- Annual SMA Conference Registration Continues to Grow!
- Registration Now Open for the 2020 Annual SMA Conference!
- Cure SMA and Genentech Partner to Provide Travel Support to Adults with SMA for the 2020 Annual SMA Conference
- 2019 SMA Researcher Meeting Summary: Basic Research Podium Presentations
- Thank You for a Wonderful 2019 Annual SMA Conference!
- 2019 SMA Researcher Meeting Summary: Special Session on Spinal and Neuromuscular Circuitry: Exploring Defects in SMA and Potential Therapeutic Targets
- The 2019 Annual SMA Conference is Here!
- The 2019 Annual SMA Conference Booklet is Now Posted Online
- Family Friendly Research Poster Session Presenters Announced
- Cure SMA to Host “Evening in the Park” at Disneyland Thanks to Support from AveXis
- Cure SMA and Genentech Partner to Provide Travel Support to Adults with SMA the Annual SMA Conference
- 2019 Annual SMA Conference Events Announced
- Cure SMA Grants More Than 900 Scholarships for Annual SMA Conference
- 2019 Annual SMA Conference Schedule is Released
- 2019 Annual SMA Conference Registration Continues to Grow!
- Community Spotlight: Amber Bosselman
- Registration is Now Open for the 2019 Annual SMA Conference!
- 2018 SMA Researcher Meeting Summary: SMA Therapy Development
- 2018 SMA Researcher Meeting Summary: Clinical Research Studies for SMA
- 2018 SMA Researcher Meeting Summary: Basic Research Podium Presentations
- 2018 SMA Researcher Meeting Special Session Summary: Clinical and Basic Questions of SMA in the Era of SMN Repletion: Challenge and Opportunity
- Thank You For a Wonderful 2018 Annual SMA Conference!
- Biogen to Present New Data at the 2018 Annual SMA Conference
- The 2018 Annual SMA Conference is Here!
- Thank You to Our 2018 Annual SMA Conference Exhibitors!
- Full Schedule of Workshops, Panels and Speakers for the 2018 Annual SMA Conference Announced
- 2018 Annual SMA Conference Booklet Released
- 2018 Family Friendly Poster Session Presenters Announced
- Registration for the 2018 Annual SMA Conference Closes in Two Weeks
- Record Number of Abstracts Submitted for 2018 SMA Researcher Meeting
- Cure SMA to Host Inaugural Clinical Care Meeting
- Adult Scholarships Offered for the 2018 Annual SMA Conference
- Registration is Now Open for the 2018 Annual SMA Conference!
- 2017 SMA Researcher Meeting Summary: Clinical Research Studies for SMA
- 2017 Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Disease Modifiers
- Victor Dubowitz Honored at 2017 Researcher Meeting for Early Contributions to the SMA Field
- 2017 Researcher Meeting Summary: Regulation of Splicing and SMN Protein Expression
- 2017 SMA Researcher Meeting Summary: SMA Therapy Development
- 2017 SMA Researcher Meeting Summary: Special Session on Fatigue and Muscle Weakness in SMA
- 2017 Annual SMA Conference Recap
- The 2017 Annual SMA Conference is Here!
- 2017 Annual SMA Conference Booklet Now Posted
- Cure SMA Announces 2017 Family Friendly Poster Session Presenters
- Record Number of Abstracts Submitted for the 2017 SMA Researcher Meeting
- Cure SMA Releases Schedule for the 2017 Annual SMA Conference
- Announcing an Evening of Wonder at Magic Kingdom Park
- Cure SMA Announces Additional Conference Support for Newly Diagnosed Families
- Adult Scholarships Offered for the 2017 Annual SMA Conference
- Family Registration for the 2017 Annual SMA Conference is Now Open
- Catch Up on Research News from the 2016 Family Friendly Research Poster Session
- 2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function
- 2016 SMA Researcher Meeting Summary: SMA Therapy Development
- 2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 2
- 2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 1
- Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene
- 2016 SMA Researcher Meeting Summary: Clinical Research Studies
- 2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA
- Cure SMA Releases 2016 Updates from Drug Programs in Clinical Trials
- Cure SMA Posts Keynote Presentations on the NDA Process
- The 2016 Annual SMA Conference is Here!
- Cure SMA Announces Biogen as Presenting Sponsor for the 2016 Annual SMA Conference
- Thank you to Our 2016 Annual SMA Conference Exhibitors!
- 2016 Annual SMA Conference Booklet Now Posted
- Announcing an Evening of Fun at Disneyland Park
- Cure SMA Releases Full Schedule for the 2016 Annual SMA Conference
- Cure SMA Announces 2016 Family Friendly Poster Session Presenters
- Annual SMA Conference to Host Panels on NDA Process and SMA Drugs in Development
- Adult Scholarships Offered for 2016 Annual SMA Conference
- Cure SMA Releases Schedule for the 2016 Annual SMA Conference
- Record Number of Abstracts Submitted for the 2016 SMA Researcher Meeting
- 2016 Annual SMA Conference on Track to Break Attendance Records
- Abstract Submission Opens for 2016 SMA Researcher Meeting
- Fall 2015 Issue of Compass Highlights SMA Researcher Meeting
- Registration Open for the 2016 SMA Researcher Meeting
- 2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 2
- 2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 1
- Family Registration for the 2016 Annual SMA Conference is Now Open
- 2015 SMA Researcher Meeting Summary: SMA Pathology
- 2015 SMA Researcher Meeting Summary: Drug Discovery
- 2015 SMA Researcher Meeting Summary: Clinical Research
- 2015 SMA Researcher Meeting Summary: Newborn Screening
- Updates from Drug Programs in Clinical Trials
- 2015 SMA Researcher Meeting Summary: Emerging Trends in Motor Neuron Pathobiology
- Seven Individuals and Families Recognized for Their Contributions to the Community
- Isis Announces Additional Trial Results at Annual SMA Conference
- Dr. Mary Schroth Joins Cure SMA as Medical Professional Education Consultant
- The 2015 Annual SMA Conference is Here!
- 2015 Annual SMA Conference Exhibitors
- 2015 Annual SMA Conference Booklet Now Posted
- Cure SMA Receives NIH Grant
- Collaborative Ways Cure SMA is Driving Research Results
- 2015 Family Friendly Poster Session Presenters Announced
- Registration for the 2015 Annual SMA Conference Closes in Two Weeks
- Annual SMA Conference to Feature Panel of Leading SMA Drug Experts
- Cure SMA Releases the Schedule for the 2015 Annual SMA Conference
- SMA Researcher Meeting to Host Over 100 Presentations
- Cure SMA Hosts SMA Researcher Meeting this June
- Registration Opens for the 2015 Annual SMA Conference
- Applications Open for 2015 Annual SMA Conference Scholarships
- 2014 SMA Researcher Meeting Summary: Clinical Trials
- 2014 SMA Researcher Meeting Summary: Therapy Development
- 2014 SMA Researcher Meeting Summary: SMA Pathology
- Join Us in Kansas City
- 2014 SMA Researcher Meeting Summary: The SMN Protein
- Catch Up on Research News from our Family Friendly Poster Session
Events & Fundraising
- Fall 2024 Walk-n-Roll Registration is Open
- Sweet Success: Baking a Difference Returns for a Second Year
- Erin Trainor Memorial Fund will Match Donations for the funding of Care Centers
- Cure SMA to Host 11th Annual Hope on the Hill Event in Washington, D.C.
- A Deliciously Easy Way to Fundraise for Cure SMA
- A Deliciously Easy Way to Fundraise for Cure SMA
- Kids: We Invite You to Bake a Difference for Cure SMA!
- Cure SMA Launches Virtual Therapy Program for Adults with SMA
- Cure SMA Career Panel Webinar Series to Launch in February
- Cure SMA Announces 2021 Summit of Strength Program Events
- Team Cure SMA Launches 2021 Race Series for Full SMA Community
- Team Biogen Runs Over 22,000 Miles During SMA Awareness Month
- 9th Annual “Hope on the Hill” Congressional Dinner Featured Dr. Francis Collins, National Institutes of Health (NIH)
- 9th Annual “Hope on the Hill” Congressional Dinner
- Team Cure SMA Raises Over $45,000 in the New York City Marathon
- Team Cure SMA Runners Brave the Rain at the Chicago Half Marathon & 5K
- 2019 Summit of Strength Program Continues its Success!
- Team Cure SMA Completes the 2019 Falmouth Road Race in Cape Cod
- Cure SMA to Host August 28th Webinar on Zolgensma Updates
- Team Cure SMA Celebrates Global Running Day!
- Team Biogen Runs from Chicago to Madison, Raising Over $12,000 for Cure SMA
- Team Cure SMA to Participate in Vermont City Marathon
- We’re Celebrating Local Support
- Team Cure SMA New York City Half Marathon Runners Raise $15,000 for Cure SMA
- The Cure SMA 2019 Goal List
- Cure SMA is Thrilled to Launch Its New and Refreshed Endurance Program: Team Cure SMA
- 8th Annual “Hope on the Hill” Congressional Dinner Will Be Held Later This Month in Washington D.C.
- Cure SMA Concert for a Cure Reaches $2 Million Milestone
- August is SMA Awareness Month!
- Summit of Strength Program Kicks Off in Cincinnati
- Cure SMA and Cytokinetics Announce National Platinum Partnership for 2018
- Cure SMA Welcomes Back AveXis as a 2018 National Premier Partner
- Cure SMA Welcomes Back Biogen as a National Premier Partner for 2018
- 7th Annual Hope on the Hill Congressional Dinner Achieves $1 Million Milestone
- Annual South Florida Gala of Hope Raises over $1 Million in 6 Years
- Help Us Shape the Future Together, and Reach our Goal of $750,000
- Registration is Now Open for the 2017 SMA Researcher Meeting
- 6th Annual South Florida Gala of Hope Inspires Familes and Supporters
- Winter 2015 Issue of Directions Now Online
- 5th Annual Congressional Dinner Brings Together Families, Government and Industry
- Great Progress, Greater Need
- 5th Annual South Florida Gala of Hope Raises Record Total of $224,000
- 5th Annual Hope on the Hill Congressional Dinner Advances SMA Advocacy
- SMART Gives $50,000 to Cure SMA to Support Research
- Western New York Teen Wins Nickelodeon Halo Award
- Spring 2015 Directions Posted Online
- High School Students Lead the Way at Concert for a Cure
- Muscles for Mckenna Gala Raises Over $100,000
- Newfield Exploration Gives Generous Grant to Cure SMA
- Cure SMA Annual Campaign Sets $1 Million Goal
- 10th Annual Grant Sheppard Memorial Scramble Reaches Milestone Year
- Bugenske Family Donates $17,000 to Cure SMA Equipment Pool
- Together, We Made History
- Help Us Make History with $750,000 for SMA Research
- Erin Trainor Memorial Fund Reaches Milestone
- Miller McNeil Woodruff Foundation Gives Generous Gift to Cure SMA
- Hope on the Hill Advances Key SMA Legislative Priorities
- Southern California Gala of Hope Brings Community and Industry Partners Together to Raise $80,000
- BAYADA Pediatrics Renews National Sponsorship with Cure SMA
- Cure SMA Honors Scott Geller with 1st Annual Community Partnership Award
- South Florida Gala of Hope Closes in on Milestone
- Cole P Randall Memorial Golf Tournament Raises $75,000
- Cure SMA Receives Generous Donation from Barney & Barney
Front Page News
- SMA Community Advocacy Wins and Champions Celebrated at Cure SMA Event
- Celebrate 40 Years of Impact – Join Us in Making a Difference!
- Spinal Muscular Atrophy (SMA) Update in Best Practices: Recommendations for Treatment Considerations
- Make an Impact with Cure SMA’s Mission, Milestones, Momentum Campaign
- Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate
- Cure SMA Hosting Puerto Rico Summit of Strength & Walk-N-Roll
- Cure SMA is Thrilled to Launch New Travel Resources Webpage and a New Travel Support Package
- New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA
- Scholar Rock Announces Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA)
- Celebrating Disability Pride and the 34th Anniversary of the ADA at the White House
- Biogen Announces Positive Topline Results from Study of Higher Dose Regimen of Nusinersen, Showing Significant Benefit in Treatment of SMA
- Community Spotlight: Viola Dwyer, SMA Community Advocate from Pennsylvania
- Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Real World Evidence Collaboration Initiatives
- Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships
- August is Spinal Muscular Atrophy (SMA) Awareness Month!
- Cure SMA Publishes Update on the Birth Prevalence of Spinal Muscular Atrophy (SMA)
- Special Session Highlights from 2024 Annual SMA Research & Clinical Care Meeting
- Congressional Action Recognizes Unmet Needs of SMA Community
- SMA Highlights: Updates from Biogen
- Clinical Research and Drug Development Session Highlights from 2024 Annual SMA Research & Clinical Care Meeting
- Spring 2024 Issue of Compass Now Available
- Thank You for an Amazing 2024 Annual SMA Conference!
- SMA Researchers and Clinicians from Around the World Gather at the 28th Annual SMA Research & Clinical Care Meeting
- Cure SMA Announces Additional $750,000 in Basic Research Funding
- Five-Year Data for Genentech’s Evrysdi Show the Majority of Treated Children With a Severe Form of SMA Achieved or Maintained the Ability to Sit, Stand or Walk
- Fall 2024 Walk-n-Roll Registration is Open
- Spinal Muscular Atrophy (SMA) Update in Best Practices: Recommendations for Diagnosis Considerations
- Novartis Gene Therapies Releases Spring SMA Community Letter
- Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Industry Collaboration Initiatives
- SMA Community Advocacy Results in Key Accessible Air Travel Win
- HHS Action Improves Healthcare Accessibility and Strengthens Protections against Disability Discrimination in Healthcare
- Genentech Releases SMA Community Letter Recapping Spring Activities
- Accessible Air Travel Update – Next Steps
- Cure SMA Launches New Resources to Enhance Clinical Trial Access
- Cure SMA Announces Expanded Phase 9 of SMA Industry Collaboration
- Biogen Releases SMA Community Letter
- Participate in the Annual Cure SMA Community Update Survey – Open Now!
- Cure SMA Awards $150,000 Grant to Lyndsay Murray,PhD, at the University of Edinburgh in Scotland
- Government Funding Bill Supports New SMA Research to Address Unmet Needs
- Cure SMA Awards $114,000 Grant to Michael Tellier, PhD, at the University of Leicester in the United Kingdom
- Apitegromab Linked to Improved Outcomes for Those with Spinal Muscular Atrophy
- Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, at The Medical College of Wisconsin
- New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy
- Novartis Presents New Data on Safety and Efficacy of Zolgensma, Including Maintained and Improved Motor Milestones in Older and Heavier Children with SMA
- Celebrate Rare Disease Day with Cure SMA
- Cure SMA Applauds DOT Proposal to Make Air Travel Safer for Wheelchair Users
- Cure SMA Awards $100,000 Grant to Elana Molotsky, PhD, at the Johns Hopkins University School of Medicine
- Cure SMA Awards $150,000 Grant to Melissa Bowerman, PhD, at Keele University
- New Report Highlights Caregiving Challenges of Individuals with Spinal Muscular Atrophy
- Cure SMA is Thrilled to Announce the Establishment of our New Chapter in Puerto Rico!
- 100% of States Now Screening Newborns for SMA
- Nevada Starts Screening for SMA – Only One State Remains!
- Analysis: Cure SMA’s Risk/Benefit Survey
- Winter Immunization Recommendations
- Novartis Gene Therapies Releases SMA Community Statement
- Genentech Releases SMA Community Letter with Evrysdi (risdiplam) Updates
- Cure SMA Featured in the NFL’s My Cause My Cleats
- Administration Officials & Members of Congress Highlight SMA Community Advocacy at 12th Annual Hope on the Hill Washington, D.C. Event
- Sweet Success: Baking a Difference Returns for a Second Year
- Biogen Shares DEVOTE and RESPOND Study Updates
- Genentech Shares 1-Year Primary Analysis from RAINBOWFISH
- Novartis Gene Therapies SMA Community Update
- Cure SMA on Capitol Hill – A Message from Allie Williams
- SMA Highlights and Updates from Biogen
- Identifying Biomarkers of Spinal Muscular Atrophy for Further Development
- NMD Pharma Initiates Phase II Trial for SMA
- Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial
- Biohaven Completes Enrollment in Pivotal Phase 3 Study
- Clinical Research and Drug Development Session Highlights from 2023 SMA Research & Clinical Care Meeting
- Genentech Releases SMA Community Letter
- Thank You for an Amazing 2023 Annual SMA Conference!
- Big Week in Washington for Cure SMA-Supported Legislation
- Cure SMA’s 2022 State of SMA Report
- Individuals with SMA Describe New Travel Hiccups and Wheelchair Damage in Their Air Travels to Cure SMA National Conference
- Luke 18:1 Foundation Donates $500,000 to Cure SMA to Support Groundbreaking SMA Research and Care
- Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA
- Congressional Committee Approves Legislation to Improve Air Travel for People with SMA and other Disabilities
- Erin Trainor Memorial Fund will Match Donations for the funding of Care Centers
- Novartis Gene Therapies Release May 2023 SMA Community Update
- Living Unlimited with Spinal Muscular Atrophy: A Booklet for Adults with SMA
- Biogen Releases Q1 2023 Community Statement
- Congress Examines Ways to Improve Air Travel for People with Disabilities
- Cure SMA Awards $75,000 Grant to Lyndsay Murray, PhD, University of Edinburgh
- Biohaven’s Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy
- District of Columbia Starts Screening for SMA During Rare Disease Month
- NEW REPORT: SMA Community Describes Air Travel Experiences as “Good, but mostly Bad, and Ugly”
- Cure SMA Awards $150,000 Grant to Florence Rage, PhD, at the Institute of Molecular Genetics of Montpellier
- Cure SMA Awards $150,000 Grant to Anton Blatnik, PhD, at The Ohio State University
- Cure SMA Supports Clinical Trial Readiness
- Cure SMA Awards $150,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires
- Cure SMA Unveils Advocacy 101 Video Series in Conjunction with 118th Congress
- Genentech Releases SMA Community Letter
- Share Your Air Travel Concerns & Experiences for Cure SMA Report to Congress
- SMA Community Progress & Advocacy Featured at Cure SMA Hope on the Hill Event
- Cure SMA to Host 11th Annual Hope on the Hill Event in Washington, D.C.
- Biogen Releases Q3 SMA Community Statement
- The Giving Season is Here!
- Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
- SMA Community Seeks SSI Improvements on Program’s 50th Anniversary
- New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA
- A Deliciously Easy Way to Fundraise for Cure SMA
- SMA Community Update from Novartis Gene Therapies
- Kids: We Invite You to Bake a Difference for Cure SMA!
- Seat Elevators on Power Wheelchairs are a Necessity, not a Luxury, according to the SMA Community
- Cure SMA Launches Benefit-Risk Survey to Assess Patient Perspective on Treatment Possibilities and Risks
- Cure SMA Care Center Network Site Expansion
- Community Statement from Novartis Gene Therapies: Zolgensma Safety Monitoring Update on Acute Liver Failure
- Biogen Shares Community Statement for SMA Awareness Month
- 2022 Cure SMA Annual Research Meeting Summaries – Special and Clinical Research Session
- 2022 Cure SMA Annual Research Meeting Summaries – Basic Research Sessions
- Cure SMA Advocacy Campaign Leads to Congressional Support for More SMA Research
- Cure SMA to Hold Patient-Led Listening Session with FDA
- Shop New Cure Merch at Our Updated Online Store
- Thank You for a Wonderful 2022 Annual SMA Conference!
- Cure SMA Attends Spring 2022 Conferences to Promote Findings from Industry Collaboration Initiatives
- Check Out Cure SMA’s State of SMA Report
- Cure SMA Announces Major Newborn Screening Milestone with Addition of Two New SMA Screening States
- SMA Community Update from Novartis Gene Therapies
- FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy
- Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships
- Cure SMA Announces Expanded Phase 7 of SMA Industry Collaboration
- Cure SMA Support Services Now Expanded to Individuals and Families in Puerto Rico
- Biohaven Licenses a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA)
- International Day of Women and Girls in Science
- Biogen Exercises Option with Ionis to Develop and Commercialize Investigational ASO for SMA
- Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With SMA
- Cure SMA Releases Updated Drug Pipeline
- Survey Alert: SMA Family Caregivers
- Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
- Cure SMA Discusses FDA-Approved SMA Treatments & Dissects Combination Therapy
- Update on Genentech/Roche Initiation of MANATEE Clinical Study
- New Data Presented at World Muscle Society 2021 Virtual Congress
- Community Statement from Biogen: New Clinical Study Planned to Evaluate the Potential Benefit of an Investigational Higher Dose of Nusinersen in People with SMA Previously Treated with Evrysdi® (risdiplam)
- Novartis Announces Lift of Partial Clinical Trial Hold, Plans to Initiate a New Study of Intrathecal Zolgensma in Older Patients with SMA
- Data for Genentech’s Evrysdi Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies with Type 1 SMA
- Cure SMA Convenes Working Group to Discuss Biomarkers for SMA
- Thank You for Another Fantastic Virtual SMA Conference!
- In Just Three Years, Cure SMA Marks Nationwide Milestone for 85% of U.S. Babies Now Screened at Birth for SMA
- Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children
- Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA
- New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA
- Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference
- Ready to Go! Platform Open for 2021 Virtual SMA Conference Events
- Cure SMA Publishes Manuscript on SMA Awareness, Screening, and Referral Patterns Amongst Pediatricians
- Cure SMA Announces 2022 Annual SMA Conference
- Community Spotlight: Doug McCullough
- Community Spotlight: Chan Family
- Cure SMA Launches New Teen & Adult Independence Assistance Package – Check It Out Today!
- Cure SMA-Funded Researchers at Ohio State University Publish Paper on Effect of Nusinersen in Adults with SMA
- Community Statement: Quarterly Update from Novartis Gene Therapies
- Community Spotlight: Elizabeth Lockwood
- Cure SMA-Funded Researchers at Columbia University Publish Paper on Fatigue in SMA
- Community Statement from Novartis Gene Therapies
- Cure SMA Attends Spring 2021 Professional Organization Conferences to Promote Findings from Industry Collaboration Initiatives
- Genentech Shares New Evrysdi Data on Improved Motor Function and Survival in Babies with SMA Type 1
- Biogen Shares New Spinraza Data Exploring Opportunities to Improve Outcomes for SMA Patients
- Participate in Annual Cure SMA Community Update Survey – Open Now!
- Cure SMA Presents Data on Cure SMA Care Center Network and SMA Clinical Data Registry
- Cure SMA Launches LifeVac Support Program to Help in Choking Event
- Scholar Rock Community Statement on TOPAZ 12-Month Topline Data
- Community Spotlight: Nick Farrell
- Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on SMA-HI, a Clinical Trial Outcome in Adults with SMA
- Nusinersen Research Updates Presented by Biogen
- Research Updates from Scholar Rock
- Community Statement from Genentech
- Community Statement from Novartis Gene Therapies
- Evrysdi Research Updates from Genentech
- Zolgensma Research Updates from Novartis Gene Therapies
- Cure SMA Publishes Quality of Life in Teens and Young Adults Manuscript in Orphanet Journal of Rare Diseases
- Biogen SMA Q1 2021 Community Statement
- Community Spotlight: I am…Viola Dwyer
- Jessica’s Story: Remembering Piper Grace
- Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM
- Cure SMA Launches Virtual Therapy Program for Adults with SMA
- Community Spotlight: I Am…Kelsey Roberts
- Cure SMA Coronavirus (COVID-19) Information Center
- Cure SMA Career Panel Webinar Series to Launch in February
- Cure SMA Announces 2021 Summit of Strength Program Events
- Cure SMA Launches Physical Therapy Webinar Series
- Cure SMA Offer Perspectives on Combination Therapy for SMA
- Team Cure SMA Launches 2021 Race Series for Full SMA Community
- Cure SMA to Host Webinar Series on Wellness for the SMA Community
- Cure SMA’s New Year Letter for 2021
- Genentech End-of-Year Community Statement
- Biogen Provides SMA Community Update on DEVOTE Study
- Community Spotlight: Brian Chiorello
- Community Spotlight: The Hoskin Family
- Cure SMA, Novartis Publish Economic Burden of SMA Analysis in Journal of Market Access & Health Policy
- Answering Questions about COVID-19 Vaccination and SMA
- Cure SMA Shares Perspective on COVID-19 Vaccination
- Community Spotlight: Nikki McIntosh
- Biogen SMA Q4 2020 Community Statement
- Now Available! New COVID-19 PPE Package from Cure SMA
- Results from Annual SMA Community Update Survey Now Published
- Cure SMA Announces Two New Virtual Programs for Adults with SMA
- Community Spotlight: Sarah Boggess, Research Coordinator, Ornamental Plant Pathology
- Summit of Strength Virtual Webinar Series Continues Its Success!
- Cure SMA Immunizations Recommendations During Flu Season and Amidst the COVID-19 Pandemic
- Scholar Rock Issues Community Statement with Update on SRK-015 in Patients with Types 2 and 3 SMA
- Community Spotlight: Perspectives from Spencer Cook, Registered Accessibility Specialist
- Community Spotlight: Kayley Shade of Sioux Falls Storm
- NeurologyLive Article Now Available, Featuring an Interview with Cure SMA’s Dr. Mary Schroth
- Cure SMA Publishes Paper in Partnership with NeurologyLive on Impact of COVID-19
- Novartis Presents New Zolgensma® Data Further Demonstrating Therapeutic Benefit
- Genentech Presents New 2-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA
- Community Spotlight: Xavier and Rose’s SMA Diagnosis
- Novartis Issues Community Statement with Update on AVXS-101 Intrathecal Clinical Development Program
- Team Biogen Runs Over 22,000 Miles During SMA Awareness Month
- Community Spotlight: Braedon’s Newborn Screening Diagnosis
- Biogen Q3 2020 Community Statement: COVID-19 Updates and Latest Milestones
- Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of SMA
- Genentech Releases Evrysdi™ FDA Approval Letter to the SMA Community
- Genentech Receives FDA Approval of Evrysdi (risdiplam) for the Treatment of SMA
- Cure SMA Launches New Medical Alert Bracelet Support Program
- Cure SMA to Meet with FDA for Critical Path Innovation Meeting
- Biogen Plans to Initiate Phase 4 Study Evaluating Benefit of Spinraza in Patients Treated with Zolgensma
- Community Spotlight: Ben Lou’s Formula for Life
- Thank You for a Wonderful 2020 Virtual SMA Conference!
- 2020 Virtual SMA Conference Booklet is Now Available Online
- Cure SMA to Fund Network of Prominent SMA Clinical Research and Treatment Centers
- Registration Now Open for the 2020 Virtual SMA Conference
- Cure SMA Announces a 2020 Virtual SMA Conference – United, At Home
- Cure SMA Announces Summit of Strength Virtual Webinar Series
- Biogen Releases Q2 Community Statement on Latest Milestones in Clinical Development Program
- Update from Scholar Rock on TOPAZ Phase 2 Clinical Trial of SRK-015 in Patients with SMA
- Genentech’s Risdiplam Shows Significant Improvement in Survival and Motor Milestones in Infants With Type 1 SMA
- Annual Cure SMA Community Update Survey Ready for Your Input
- Cure SMA Launches New COVID-19 Support Package
- COVID-19 Assistance Program Available to SMA Community
- Grant Announcement: Cure SMA Awards $190,000 Grant to Stephen Meriney, Ph.D., University of Pittsburgh
- Cure SMA Announces Cancellation of 2020 Annual SMA Conference and Research & Clinical Care Meeting
- Community Statement from AveXis Regarding Commitment to SMA Community Amid COVID-19 Pandemic
- Community Statement from Genentech on Risdiplam Approval Timeline
- Answering Your Coronavirus (COVID-19) Medical Questions
- Biogen Announces First Patient Treated With Higher Dose Of Spinraza In Phase 2/3 DEVOTE Study
- Zolgensma® Data Shows Rapid, Significant, Clinically Meaningful Benefit in SMA, Including Prolonged Event-free Survival, Motor Milestone Achievement and Durability, Now Up to 5 Years Post-dosing
- Cure SMA Launches Part 2 of “Spotlight on SMA” in Partnership with Neurology Reviews
- Cure SMA Announces Additional Event Cancellations; No Changes to Plans and Recommendations for Annual SMA Conference
- 2020 Annual SMA Conference Schedule is Released
- Grant Announcement: Cure SMA Awards $150,000 Grant to Emily Welby, Ph.D., Medical College of Wisconsin
- Biogen Issues Community Letter with Perspective on Coronavirus (COVID-19)
- Grant Announcement: Cure SMA Awards $190,000 Grant to Umrao Monani, Ph.D., Columbia University
- Cure SMA Announces Event Cancellations, Recommendations on Coronavirus, and Update on Annual SMA Conference
- Cure SMA Awards $100,000 Grant to Rashmi Kothary, Ph.D., Ottawa Hospital Research Institute
- Grant Announcement: Cure SMA Awards $150,000 Grant to Timra Gilson, Ph.D., Indiana University
- Grant Announcement: Cure SMA Awards $150,000 Grant to Dmytro Morderer, Ph.D., Mayo Clinic, Jacksonville, FL
- Community Spotlight: Cesar Rodrigues
- Grant Announcement: Cure SMA Awards a $170,000 Grant to Lyndsay Murray, Ph.D., University of Edinburgh
- Cure SMA Working Group Revises Recommendations for the Treatment of Infants Diagnosed with SMA via Newborn Screening Who Have 4 Copies of SMN2
- Cure SMA Launches “Spotlight on SMA” Partnership with Neurology Reviews
- Celebrate Rare Disease Day 2020 with Cure SMA
- Lights…Camera…Action! Search Is On for Child with SMA to Appear in NBC Television Pilot
- Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 Years with Type 2 or Type 3 SMA
- Cure SMA Adds New Equipment Item for Teens and Adults with SMA
- Summit of Strength Program Dates Confirmed
- Genentech’s Risdiplam Meets Primary Endpoint in Pivotal FIREFISH Trial in Infants with Type 1 SMA
- Annual SMA Conference Registration Continues to Grow!
- Community Statement from Scholar Rock Regarding SRK-015
- Cure SMA and Biogen to Co-Host Webinar on DEVOTE Clinical Trial – Register Today!
- Cure SMA’s New Year Letter for 2020
- Community Spotlight: The Forcier Family
- Community Spotlight: Scurria Family
- A Look Back at 2019: Cure SMA Research Year-in-Review
- Cure SMA Awards Grants to Four States to Expediate the Implementation of SMA Newborn Screening
- Biogen Issues Q4 2019 Community Statement on Spinraza
- Community Spotlight: The Veit Family
- 9th Annual “Hope on the Hill” Congressional Dinner Featured Dr. Francis Collins, National Institutes of Health (NIH)
- Fall 2019 Issue of Compass Now Available
- Registration Now Open for the 2020 Annual SMA Conference!
- Update on Cure SMA Summit of Strength Program – Gearing Up for 2020!
- Check Out the Cure SMA Booklet on “Scientific Considerations for Drug Combinations”
- 9th Annual “Hope on the Hill” Congressional Dinner
- Cure SMA and Genentech Partner to Provide Travel Support to Adults with SMA for the 2020 Annual SMA Conference
- Community Statement from Genentech: FDA Grants Priority Review to Risdiplam for Spinal Muscular Atrophy
- SMA Concordance Among Siblings Published in the Journal of Neuromuscular Diseases
- Caring for the Caregiver by Brynne Willis
- Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy
- Team Cure SMA Raises Over $45,000 in the New York City Marathon
- Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SUNFISH Trial in People With Type 2 or 3 Spinal Muscular Atrophy
- A Tiny Warrior: Winnie Stanley’s Story
- Community Spotlight: Angela Wrigglesworth
- AveXis Issues Community Statement on AVXS-101 Clinical Hold
- Novartis Announces AVXS-101 Intrathecal Study Update
- Community Spotlight: Steven Verdile
- Cure SMA Establishes an Adult Advisory Council
- Cure SMA and Ionis Announce Holiday Card Contest
- Team Cure SMA Runners Brave the Rain at the Chicago Half Marathon & 5K
- AveXis Presents Updated STRONG Data Demonstrating a Higher Mean Increase in Hammersmith Functional Motor Scale-Expanded (HFMSE) Scores Among Older SMA Type 2 Patients Following One-time Intrathecal Administration of AVXS-101
- Scholar Rock to Present Preclinical and Phase 1 Clinical Data on SRK-015 at the World Muscle Society Congress
- Landmark NURTURE Study of Infants with Spinal Muscular Atrophy (SMA) Treated Pre-Symptomatically with Spinraza Published in Neuromuscular Disorders
- Risdiplam Data Presented at World Muscle Society Congress Demonstrates Continued Benefit for Patients with Spinal Muscular Atrophy
- AveXis Presents New Data at EPNS Continuing to Show Significant Therapeutic Benefit of Zolgensma® in Prolonging Event-free Survival Now up to 5 Years of Age in Patients with SMA Type 1
- 2019 Summit of Strength Program Continues its Success!
- Cure SMA Newborn Screening Registry (NBSR)
- Zolgensma Update and Approval Webinars Now Available Online
- 2019 Researcher Meeting Summary: Clinical Research Studies for SMA
- Team Cure SMA Completes the 2019 Falmouth Road Race in Cape Cod
- 2019 SMA Researcher Meeting Summary: Basic Research Podium Presentations
- Thank You for a Wonderful 2019 Annual SMA Conference!
- Cure SMA Awards Grants to Four States to Expediate the Implementation of SMA Newborn Screening
- 2019 SMA Researcher Meeting Summary: Special Session on Spinal and Neuromuscular Circuitry: Exploring Defects in SMA and Potential Therapeutic Targets
- Cure SMA Update on Data Accuracy Issues with AveXis’ SMA Therapy, Zolgensma
- Cure SMA Releases Status Update on Data Accuracy Issues with Zolgensma
- FDA Issues Statement on Data Accuracy Issues with Recently Approved Gene Therapy
- Biogen Issues Community Statement on Spinraza
- SMA Awareness Month 2019 Has Begun!
- Biogen Announces New NURTURE Data at the 2019 Annual SMA Conference
- Updated SMA Drug Pipeline Now Available
- Cytokinetics Releases New Data from Reldesemtiv Clinical Study at the 2019 Annual SMA Conference
- The 2019 Annual SMA Conference is Here!
- Cure SMA Receives $150,000 Grant From the Oscar G and Elsa S Mayer Family Foundation
- Community Spotlight: Laura Watson
- The 2019 Annual SMA Conference Booklet is Now Posted Online
- Family Friendly Research Poster Session Presenters Announced
- Biogen Issues Q2 Community Statement on Spinraza
- Team Cure SMA Celebrates Global Running Day!
- Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers
- Cure SMA to Host “Evening in the Park” at Disneyland Thanks to Support from AveXis
- AveXis and Cure SMA to Co-Host Webinar on the FDA’s Approval of Zolgensma
- AveXis Issues Community Statement on Zolgensma Approval
- AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age
- Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado
- Novartis Issues Community Statement on Branaplam Clinical Study
- Team Biogen Runs from Chicago to Madison, Raising Over $12,000 for Cure SMA
- Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA
- Spring 2019 SMA Newborn Screening Update
- Team Cure SMA to Participate in Vermont City Marathon
- Dr. Arthur Burghes Named 2019 Distinguished Scholar
- Scholar Rock Issues Community Statement on the Initiation of a New Clinical Trial for SRK-015
- Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in Spinal Muscular Atrophy at the AAN Annual Meeting
- AveXis Presents Data at AAN Annual Meeting Demonstrating Efficacy of Zolgensma in Broad Spectrum of Spinal Muscular Atrophy (SMA) Patients
- Cure SMA and Genentech Partner to Provide Travel Support to Adults with SMA the Annual SMA Conference
- Biogen to Present Data at AAN Highlighting Spinraza and Emerging Biomarkers for SMA
- Genentech Presents New Data at the 2019 AAN Annual Meeting
- Community Spotlight: Kyle and Laura Derkowski
- Biogen Presents New Data Regarding Adults and Infants with SMA
- Community Update Survey Q&A
- 2019 Annual SMA Conference Events Announced
- Cure SMA Grants More Than 900 Scholarships for Annual SMA Conference
- Cure SMA Care Center Network Expands to 10 Sites
- 2019 Annual SMA Conference Schedule is Released
- 2019 Annual SMA Conference Registration Continues to Grow!
- Cure SMA Awards $150,000 Grant to Jocelyn Cote, PhD, University of Ottawa
- What is the Summit of Strength Program?
- We’re Celebrating Local Support
- Cure SMA Awards $200,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires
- Cure SMA Awards $200,000 Grant to Charlotte Sumner, MD, Johns Hopkins University
- The Winter 2019 issue of Compass in Now Available Online
- Team Cure SMA New York City Half Marathon Runners Raise $15,000 for Cure SMA
- Record Number of Abstracts Submitted for the 2019 SMA Researcher Meeting
- Cure SMA and Parent Project Muscular Dystrophy Announce Collaboration
- Cure SMA Launches Third Annual Community Survey to Address Important Issues in SMA Treatment
- Recording of Genentech/Roche and Cure SMA Webinar Now Available Online
- Cure SMA Advocates for SMA Community at ICER Hearing
- Biogen Provides 2019 Community Update on Spinraza
- Genentech/Roche and Cure SMA to Co-Host Webinar
- New Dates and Locations Announced for 2019 Summits of Strength
- Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans
- Community Spotlight: Kristen Smith
- Arkansas and Florida Adopt SMA Newborn Screening
- Cure SMA Awards $150,000 Grant to Veronica Pessino, PhD, Salk Institute for Biological Sciences
- Community Spotlight: Amber Bosselman
- Show Your Rare. Show You Care.
- Cure SMA Awards $150,000 Grant to Laxman Gangwani, PhD, Texas Tech University
- Register for Cure SMA’s Advocacy 101 Webinar: Learn How to Make a Difference
- The Cure SMA 2019 Goal List
- Cure SMA’s 2019 Commitments to the Community
- Cytokinetics Announces Receipt of FDA Feedback Regarding Reldesemtiv in Patients with SMA
- Cure SMA is Thrilled to Launch Its New and Refreshed Endurance Program: Team Cure SMA
- Be A Part of Cure SMA’s Valentine’s Day Advocacy Campaign and Support the Newborn Screening Saves Lives Reauthorization Act
- Heather Tomko on Goal-Setting for the New Year
- Fall 2018 Issue of Directions Now Available
- Congratulations to the Winners of Cure SMA’s Video Contest!
- Community Spotlight: Rebecca Smith and Micah Biello
- Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam
- Teen and Young Adult Survey Extended Through January 21st
- Community Spotlight: The Jankowski Family
- Cure SMA Announces Expanded Phase 3 of SMA Industry Collaboration
- Hope on the Hill Congressional Dinner Raises $200,000
- December Webinar on SMA Treatments and Clinical Trials Now Available Online
- 2018 Research Year-in-Review
- Cure SMA Funding Leads to 16 New Research Publications in 2018
- Community Spotlight: The Berkovits Family
- Advocacy Year-in-Review 2018
- Biogen Issues Q4 Community Statement on Spinraza
- AveXis Issues Community Statement on BLA Acceptance
- Closing out 2018 with Good News: Virginia Adopts SMA to their Newborn Screening Panel, Arizona Moves Closer to Adopting SMA Newborn Screening and Illinois Medicaid Covers Spinraza Treatment
- Fall 2018 Issue of Compass Now Available
- Novartis Announces FDA Filing Acceptance and Priority Review for AVXS-101
- Register for Cure SMA’s Webinar on SMA Treatments and Clinical Trials
- Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of SMA
- Cure SMA Launches “SMArt Moves” for Early Diagnosis and Treatment
- 8th Annual “Hope on the Hill” Congressional Dinner Will Be Held Later This Month in Washington D.C.
- Cure SMA is Thrilled to Launch a New Support Program for Teens and Adults with SMA
- Summit of Strength Program Receives Positive Feedback and Increases Attendance
- Registration is Now Open for the 2019 Annual SMA Conference!
- Kansas Adopts SMA Newborn Screening as Several Other States Move Closer to Adoption
- Meet the NDEAM Webinar Panelists
- Meet the National Disability Employment Awareness Month (NDEAM) Panelists
- NDEAM Spotlight: Jaclyn Greenwood
- National Disability Employment Awareness Month Spotlight: Kim Hill
- Register for Cure SMA’s First National Disability Employment Awareness Month Webinar
- AveXis Issues Community Statement on FDA Filing for SMA Type I
- AveXis Files for FDA Approval of Gene Therapy for Spinal Muscular Atrophy
Type I - North Carolina to Screen Newborns for SMA Through Early Check
- Breakthrough Prize Awarded to Adrian Krainer and Frank Bennett for SMA Research Leading to Spinraza
- Cure SMA Receives Generous Funding from Luke 18:1 Foundation
- SMA Care Center Network and Clinical Data Registry Launched
- New Spinraza Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with SMA
- Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA
- Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress
- Genentech Releases Community Statement on Risdiplam
- Community Spotlight: Adrienne Vollmer
- Cure SMA Launches a Family Support App for Your Mobile Device!
- Community Spotlight: Allyson Henkel
- Community Spotlight: The Lasko Family
- Cure SMA’s Approach to Newborn Screening
- SMA Newborn Screening Advancements
- New York to Implement Newborn Screening for SMA October 1st
- Sweet Baby Zane Funds New Equipment Pool Item
- Cure SMA Receives Generous Donation from the Cash Scanlon-Philips Foundation
- Cure SMA Provides Funding for “Patient Reported Impact of Symptoms in Spinal Muscular Atrophy (PRISM-SMA)” Published in Neurology
- Community Spotlight: The Anton Jensen Family
- Community Spotlight: The Nelson Family
- Community Spotlight: The Zmaczynski Family
- Community Spotlight: Doug McCullough
- Cure SMA Concert for a Cure Reaches $2 Million Milestone
- Community Spotlight: The Colone Family
- Updated 2018 SMA Standards of Care Statements Available Online
- Illinois Adopts Permanent SMA Screening
- Heather Tomko Crowned as Ms. Wheelchair USA 2018
- 2018 SMA Researcher Meeting Summary: SMA Therapy Development
- Community Spotlight: Brynne Willis
- August is SMA Awareness Month!
- 2018 SMA Researcher Meeting Summary: Clinical Research Studies for SMA
- 2018 SMA Researcher Meeting Summary: Basic Research Podium Presentations
- 2018 SMA Researcher Meeting Special Session Summary: Clinical and Basic Questions of SMA in the Era of SMN Repletion: Challenge and Opportunity
- Wisconsin Moves One Step Closer to SMA Newborn Screening
- Summit of Strength Program Kicks Off in Cincinnati
- Cure SMA Awards $300,000 Grant to Kevin Hodgetts, PhD, The Brigham and Women’s Hospital
- Dr. John Kissel, Chair of Neurology at OSU Wexner Medical Center, Retires After 40 Years
- Roche Releases Community Update on Risdiplam
- Cure SMA Awards $300,000 Grant to Umrao Monani, PhD, Columbia University
- HHS Secretary, Alex Azar, Recommends Nationwide Newborn Screening for SMA
- SMA Newborn Screening Moves Forward in New Jersey
- Thank You For a Wonderful 2018 Annual SMA Conference!
- Summer 2018 Issue of Compass Now Available
- PTC Therapeutics Announces Risdiplam (RG7916) is Well Tolerated at All Dose Levels With No Drug-Related Safety Findings
- Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference
- Biogen to Present New Data at the 2018 Annual SMA Conference
- The 2018 Annual SMA Conference is Here!
- Thank You to Our 2018 Annual SMA Conference Exhibitors!
- Traveling with SMA
- Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015
- Ohio Moves One Step Closer to SMA Newborn Screening
- Roche Releases Community Statement on Olesoxime Program
- Full Schedule of Workshops, Panels and Speakers for the 2018 Annual SMA Conference Announced
- Cure SMA Awards $150,000 Grant to Kathryn Swoboda, MD, Massachusetts General Hospital
- 2018 Annual SMA Conference Booklet Released
- Biogen Releases Community Statement on Spinraza Access and New Data
- 2018 Family Friendly Poster Session Presenters Announced
- Spring 2018 Issue of Directions Now Available
- Cure SMA and Cytokinetics Announce National Platinum Partnership for 2018
- Indiana Becomes Fourth State to Adopt Newborn Screening for Spinal Muscular Atrophy
- SMA Advocates Meet With House and Senate Offices
- Cure SMA Awards $150,000 Grant to Chad Heatwole, MD, University of Rochester
- Uniting the SMA Community Through Local Programs and Support
- Invitae Partners with Biogen to Offer Free Genetic Testing for SMA
- Dr. Richard Finkel, M.D., Honored with the American Academy of Neurology’s 2018 Sidney Carter Award in Child Neurology
- AveXis to Present Phase 1 Data at the 70th American Academy of Neurology Annual Meeting
- SMA Community Leaders to Gather in Washington DC to Network, Learn and Advocate
- The Discovery of Spinraza
- There is Still Time to Complete Cure SMA’s 2018 Community Survey
- Biogen Presents New Data at the American Academy of Neurology (AAN) Annual Meeting
- Spring 2018 Issue of Compass Now Available Online
- Registration for the 2018 Annual SMA Conference Closes in Two Weeks
- Genentech to Present New RG7916 Data at the American Academy of Neurology (AAN) Annual Meeting
- Cure SMA Awards $150,000 Grant to Bakri Elsheikh, MD, Ohio State University Wexner Medical Center
- Cure SMA Database Findings Published in the Journal of Neuromuscular Diseases
- Novartis Agrees to Acquire AveXis
- SMA Newborn Screening Guidelines Published in Journal of Neuromuscular Diseases
- Community Spotlight: Nathan Yates
- Cure SMA Welcomes Back AveXis as a 2018 National Premier Partner
- Cure SMA Awards $150,000 Grant to Brunhilde Wirth, PhD, Institute of Cologne, Germany
- Cure SMA Awards $50,000 Grant to Rashmi Kothary, PhD, University of Ottawa
- Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with SMA
- Cure SMA Awards $150,000 Grant to Robin Parks, PhD, University of Ottawa
- Cure SMA Welcomes Back Biogen as a National Premier Partner for 2018
- House and Senate Vote to Increase Federal Funding for Newborn Screening Programs
- Cure SMA Awards $150,000 Grant to Charlotte Sumner, MD, Johns Hopkins University
- Record Number of Abstracts Submitted for 2018 SMA Researcher Meeting
- The Discovery of SMA
- Roche Releases Community Statement on RG7916 and Olesoxime Programs
- Cure SMA Awards $75,000 Grant to Robert Kalb, MD, Northwestern University
- Cure SMA to Host Inaugural Clinical Care Meeting
- Cure SMA Awards $150,000 Grant to Umrao Monani, PhD, Columbia University
- Biogen Announces New Interim Phase 2 Results From NURTURE
- Cure SMA Launches Second Annual Community Survey to Address Important Issues in SMA Treatment
- Indiana is the Fourth State to Adopt Permanent SMA Screening
- Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, Medical College of Wisconsin
- Awareness is Best Raised When We Work Together
- Cure SMA Welcomes New Medical Advisory Council Committee Members
- SMA Newborn Screening Moves Forward in Indiana, Ohio and Georgia
- #ShowYourRare and Raise SMA Awareness on World Rare Disease Day
- Last Week’s Webinar on Treatment Access and Clinical Trials Now Available
- Cure SMA Receives Generous Gift from the Weisman Family Foundations
- Phase 2 Clinical Trial Results for CK-2127107 Expected in Second Quarter of 2018
- Cure SMA Receives Generous Gift
- Biogen Releases Statement on CHERISH Data and Plans for New Clinical Research
- Additional Funding for SMA Care Centers Announced
- Cure SMA Announces Local Educational Symposiums in 16 US Locations
- Winter 2018 Compass Now Available Online
- Advisory Committee on Heritable Disorders in Newborns and Children Recommends Nationwide Newborn Screening for Spinal Muscular Atrophy
- Utah Becomes First State to Implement Permanent Screening for SMA
- Federal Committee to Vote on Spinal Muscular Atrophy Newborn Screening on Thursday
- Preliminary Data from FIREFISH Trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy
- AveXis Releases Community Statement on Expanded Clinical Trials
- Cure SMA to Host Webinar on Treatment Access and Clinical Trials on February 15
- SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report
- 2018 Updated SMA Drug Pipeline Released
- Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials
- Cure SMA Launches Advocacy Action Network
- Minnesota Becomes Second State to Adopt Newborn Screening for SMA
- Cure SMA Announces Funding for Care Centers Across the US
- Biogen and Ionis Enter Into New Collaboration to Identify Novel Therapies for the Treatment of Spinal Muscular Atrophy
- Cure SMA Assists Hundreds of Families in 2017
- Miller McNeil Woodruff Foundation Awards Cure SMA Generous Gift
- Fall 2017 Directions Now Available for Download
- 2017 Research Year-in-Review
- Cure SMA Advocates Continue to Advance Newborn Screening Efforts
- AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
- Adult Scholarships Offered for the 2018 Annual SMA Conference
- Cure SMA-Funded Research Results in 21 Published Journal Articles
- Cytokinetics Publishes Clinical Trial Data for CK-2127107
- Cure SMA-Funded Researcher, Stephen Kolb, Publishes Paper
- 7th Annual Hope on the Hill Congressional Dinner Achieves $1 Million Milestone
- SMA Advocates Testify at Federal Newborn Screening Meeting
- Biogen Issues Community Update on Spinraza
- 2017 World Muscle Society Congress Recap
- New Clinical Trial Data Published in New England Journal of Medicine
- Fall 2017 Issue of Compass Now Available
- Cure SMA Launches Benefit-Risk Survey to Address Important Issues in Treatment Access and Future Drug Approvals in SMA
- Cure SMA Announces Over $5 Million in New Research and Care Funding
- Registration is Now Open for the 2018 Annual SMA Conference!
- Cure SMA Meets in Utah with State and Federal Elected Officials to Support Newborn Screening
- Genentech/Roche Releases Clinical Trial Update for SUNFISH (RG7916)
- Recording of the September Webinar on Spinraza Updates Now Available
- AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process
- Cure SMA Contributes Article to a Special Issue of the Journal Gene Therapy
- Newborn Screening Advocacy Update
- Novartis Releases Update on LMI070 (Branaplam) Clinical Trial
- Applications for Cure SMA Medical Advisory Council Due October 15th
- Genentech/Roche Releases Clinical Trial Update for RG7916
- 2017 SMA Awareness Month Recap
- Cure SMA to Host Webinar Updating the Community on Spinraza Access
- 2017 SMA Researcher Meeting Summary: Clinical Research Studies for SMA
- 2017 Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Disease Modifiers
- Victor Dubowitz Honored at 2017 Researcher Meeting for Early Contributions to the SMA Field
- SMA Documentary Premieres at 2017 Rhode Island International Film Festival
- Cure SMA-Funded Researchers, Diane Murrell and Deborah Boroughs, Publish Papers
- 2017 Researcher Meeting Summary: Regulation of Splicing and SMN Protein Expression
- Centers for Medicare and Medicaid Services (CMS) Survey
- Roche Opens US Recruitment for Phase 2 Clinical Trial
- SMA Families and Cure SMA Staff Visit Capitol Hill for Advocacy Efforts
- 2017 SMA Researcher Meeting Summary: SMA Therapy Development
- 2017 SMA Researcher Meeting Summary: Special Session on Fatigue and Muscle Weakness in SMA
- Cure SMA Receives Donation from the Bugenske Family
- Cure SMA Arkansas Chapter Announced
- August is SMA Awareness Month!
- Biogen Releases Update on Spinraza Access
- Summer 2017 Issue of Compass Now Available
- Call to Action: Senate Expected to Vote on Healthcare Bill Today
- 2017 Annual SMA Conference Recap
- Missouri is the First State to Institute Newborn Screening for Spinal Muscular Atrophy
- Cure SMA Launches Newborn Screening Grassroots Advocacy Campaign for Spinal Muscular Atrophy Newborn Screening
- Summer 2017 Update to SMA Drug Pipeline is Released
- Cure SMA Funded Standard of Care Paper Published in Neuromuscular Disorders Journal
- The 2017 Annual SMA Conference is Here!
- Call-to-Action: Senate Health Care Bill Released and Vote Expected Next Week
- Cure SMA Update – Senate Healthcare Bill
- Launch of Phase 2 SMA Industry Collaboration Announced
- 2017 Annual SMA Conference Booklet Now Posted
- SMA Newborn Screening Moves Forward in Missouri and Florida
- AveXis Announces Alignment with the FDA on Company’s GMP Commercial Manufacturing Process for AVXS-101
- Cure SMA Advocacy Activities for the 2017 Year-to-Date
- Update: President’s Budget Proposes Significant Cuts to Federal Health Programs That Are Critically Important to SMA Patients and Families
- Anthem Updates Insurance Policy for Spinraza
- European Commission Grants Marketing Authorization for Spinraza, Making it First Approved SMA Treatment in the European Union
- Cure SMA Awards $140,000 Grant to Oliver Gruss, PhD, Rheinische Friedrich-Wilhelms-Universitat Bonn and Utz Fischer, PhD, Julius-Maximilians-Universitat Wurzburg, Germany
- Cytokinetics Receives FDA Orphan Drug Designation for CK-2127107 for SMA
- Cure SMA Announces 2017 Family Friendly Poster Session Presenters
- Cure SMA Awards $140,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires, Argentina
- RUSP Nomination for SMA Accepted into Evidence Review
- Cure SMA Welcomes Back Biogen as a National Premier Partner for 2017
- Cure SMA Awards $75,000 Grant to Yong-Chao Ma, PhD, Ann & Robert H. Lurie Children’s Hospital of Chicago
- Cure SMA Awards $75,000 Grant to Stephen J. Kolb, PhD, Ohio State University
- Cure SMA Awards $75,000 Grant to Chris Lorson, PhD, University of Missouri
- Cure SMA Announces Increase in Basic Research Funding, Now Exceeding $1 Million in Grants
- Update on the Passage of the AHCA
- Cure SMA Grassroots Alert – AHCA
- Cure SMA Awards $140,000 Grant to Christine DiDonato, PhD, Ann & Robert H. Lurie Children’s Hospital of Chicago
- Biogen Releases New Data from Cherish and Nurture Trials
- SMA Community’s Voice Heard “Loud and Clear” at Last Week’s Patient Focused Drug Development Meeting with the FDA
- Cure SMA to Host Webinar on Spinraza Access, as List of Administration Sites is Released
- The European Medicines Agency (EMA) Recommends Granting Marketing Authorization in the European Union for Spinraza
- Cure SMA Awards $75,000 Grant to Jean Giacomotto, PhD, University of Queensland, Australia
- Cure SMA Publishes Second Journal Article on the Patient Voice
- Cure SMA Awards $30,000 Grant to Remy Bordonne, PhD, CNRS-Centre National de Recherche Scientifique, France
- Cure SMA Receives Generous Donations from Fighting for Kaiden Foundation
- Record Number of Abstracts Submitted for the 2017 SMA Researcher Meeting
- Cytokinetics Announces Start of Second Cohort in Phase 2 Clinical Trial of CK-2127107 in Patients with SMA
- Cure SMA Coverage and Payment Policy Project Continues to Move Forward
- Genentech Roche Releases Community Letter with Clinical Trial Updates
- Cure SMA Releases Schedule for the 2017 Annual SMA Conference
- Cure SMA Awards $140,000 Grant to Joceyln C̫t̩, PhD, University of Ottawa
- American College of Obstetricians and Gynecologists Recommends Expanded SMA Carrier Screening
- AveXis Reports Results from Phase 1 Trial of AVXS-101
- Know Your Resources: How Congressional Caseworkers Can Help You Obtain Insurance Coverage for New Therapies
- Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University
- Cure SMA Releases Care Series Booklet on Insurance
- SMA is Submitted to the Federal Newborn Recommended Uniform Screening Panel (RUSP)
- Announcing an Evening of Wonder at Magic Kingdom Park
- Cure SMA to Host Webinar with the FDA and Patient Advocates
- Registration Opens for the SMA Patient-Focused Drug Development Meeting with the FDA
- Spinraza Access Update Webinar
- Cure SMA Launches Community Survey to Address Urgent and Important Issues in SMA Treatment
- Biogen Presents New ENDEAR Data Showing SPINRAZA (nusinersen) Significantly Reduces Risk of Death or Permanent Ventilation
- Updated SMA Drug Pipeline Released as First Commercial Patient Receives Spinraza
- SMA Patient-Focused Drug Development Meeting with FDA Set for April 18, 2017
- Cure SMA Assists Hundreds of Families in 2016
- Cure SMA Funding Results in 16 New Research Publications
- 2016 Research Year-in-Review: An Historic Year for Our Community
- Biogen Resources for the SMA Community
- Join Us for an Upcoming Webcast about SPINRAZA TM (nusinersen)
- Biogen Provides Community Statement and Q&A on the Approval of Spinraza for SMA
- Ionis Pharmaceuticals Issues Community Statement on the Approval of Spinraza
- FDA Approves Spinraza for SMA
- Cure SMA Participates in a Special FDA Listening Session
- Cure SMA Announces Additional Conference Support for Newly Diagnosed Families
- SMA Community Conducts Outreach Visits with Members of Congress
- The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants
- The Miller McNeil Woodruff Foundation Makes a Generous Contribution to Cure SMA
- Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on Clinical Trial Outcomes in Adults with SMA
- Annual Congressional Dinner Brings SMA Community Together on the Eve of 21st Century Cure Bill Passage
- Annual South Florida Gala of Hope Raises over $1 Million in 6 Years
- Help Us Shape the Future Together, and Reach our Goal of $750,000
- Over 35 Cure SMA-Funded Researchers Contribute to Comprehensive SMA Encyclopedia
- Adult Scholarships Offered for the 2017 Annual SMA Conference
- Registration is Now Open for the 2017 SMA Researcher Meeting
- Cure SMA Launches Insurance Coverage and Payment Policy Project
- 6th Annual South Florida Gala of Hope Inspires Familes and Supporters
- Cure SMA Joins SMA Europe and TREAT-NMD for Meeting with EMA Regulators
- Cure SMA to Host Webinar on the New Drug Application Process
- Biogen and Ionis Community Statement on Interim Analysis of CHERISH (Type 2) Trial
- Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3 CHERISH Study
- AveXis Announces Single-Arm Design for Pivotal Study of AVXS-101
- Family Registration for the 2017 Annual SMA Conference is Now Open
- Spotlight Innovation Launches Development of STL-182 to Treat SMA
- 6th Annual Congressional Dinner Furthers SMA Advocacy Work
- Cure SMA-funded Researcher Sara Custer Publishes Paper
- SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA
- Cure SMA and Ionis Announce 2016 Holiday Card Contest
- AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 at the World Muscle Society Congress
- Biogen Presents New Data at World Muscle Society Congress
- Cure SMA Announces Newborn Screening Initiative in Partnership with MDA
- Biogen and Ionis Release Community Statement on NDA Filing Completion
- Roche Launches New Phase 2 Clinical Trials
- Catch Up on Research News from the 2016 Family Friendly Research Poster Session
- Roche Provides New Update on Clinical Development of RG7916
- 2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function
- Cure SMA Welcomes Aboard New Board and Committee Members
- 2016 SMA Researcher Meeting Summary: SMA Therapy Development
- Summer 2016 Issue of Compass Now Available
- 2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 2
- 2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 1
- Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene
- 2016 SMA Researcher Meeting Summary: Clinical Research Studies
- Cure SMA Plans a Series of Educational Modules for the SMA Community
- Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy
- AveXis Reports Second Quarter 2016 Interim Data from Ongoing Phase 1 Trial of AVXS-101 (Gene Therapy)
- 2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA
- Novartis Releases Community Update on the Study of LMI070
- Clinical Trial of CK-2127107 in Patients with Spinal Muscular Atrophy
- Biogen and Ionis Pharmaceuticals Provide Important Update on First Ever SMA Regulatory Filings
- Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA
- Cure SMA Announces $2.5 Million in New Planned Research Funding
- AveXis Receives FDA Breakthrough Therapy Designation for AVXS-101 (Gene Therapy) for SMA
- Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports
- Cure SMA Awards $50,000 Drug Discovery Grant to Barrington Burnett, PhD, at Uniformed Services University of the Health Sciences
- Cure SMA Releases 2016 Updates from Drug Programs in Clinical Trials
- Cure SMA Posts Keynote Presentations on the NDA Process
- Cure SMA Funds $2 Million in New Research in 2015-2016
- The 2016 Annual SMA Conference is Here!
- Cure SMA Awards $304,000 Drug Discovery Grant to Livio Pellizzoni, PhD, at Columbia University
- Cure SMA Receives 2016 NIH Grant
- Cure SMA Announces Biogen as Presenting Sponsor for the 2016 Annual SMA Conference
- Cure SMA Releases June 2016 Update to the SMA Drug Pipeline
- Thank you to Our 2016 Annual SMA Conference Exhibitors!
- Biogen and Ionis Release Spring 2016 Community Update
- 2016 Update on SMA-FDA Interactions
- 2016 Annual SMA Conference Booklet Now Posted
- Update on the Clinical Development of LMI070
- Spring 2016 Issue of Compass Now Available
- Cure SMA Awards $300,000 Drug Discovery Grant to Charlotte Sumner, in Collaboration with Imago Pharmaceuticals
- Announcing an Evening of Fun at Disneyland Park
- Cure SMA Releases Full Schedule for the 2016 Annual SMA Conference
- Cure SMA Awards $70,000 Grant to Christine DiDonato, Lurie Children’s Hospital of Chicago
- AveXis Reports Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1
- Cure SMA Announces 2016 Family Friendly Poster Session Presenters
- Cure SMA Welcomes New Board and Committee Members
- Ionis Releases Data Update from Phase 2 Study in Infants with SMA
- Cure SMA and the SMA Community Featured by Astellas Pharmaceuticals
- Annual SMA Conference to Host Panels on NDA Process and SMA Drugs in Development
- Cure SMA-Funded Researchers Receive NIH Grant
- Adult Scholarships Offered for 2016 Annual SMA Conference
- Global Genes Features Cure SMA in Patient Voice Project
- Cure SMA Awards $30,000 Grant to Jocelyn Cote, PhD, University of Ottawa
- Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University
- Cure SMA Awards $90,000 Grant to Antoine Cl̩ry, PhD, ETH Zurich
- Cure SMA Awards $140,000 Grant to Christine Beattie, PhD, Ohio State University
- Cure SMA Releases Schedule for the 2016 Annual SMA Conference
- AveXis Releases New Data on Phase 1 Clinical Trial
- Record Number of Abstracts Submitted for the 2016 SMA Researcher Meeting
- Senate HELP Committee Passes Another 7 Innovation Bills
- Winter 2015 Issue of Directions Now Online
- Cure SMA-Funded Researchers Publish Paper on Motor Neuron and Astrocyte Defects
- 2016 Annual SMA Conference on Track to Break Attendance Records
- Cure SMA Awards $140,000 Grant to Rashmi Kothary, MD, Ottawa Hospital Research Institute
- Cure SMA Awards $140,000 Grant to Megerditch Kiledjian, PhD, of Rutgers
- Senate HELP Committee Passes Innovation Bills
- Update on 21st Century Cures/Innovation: the Senate’s “Step-by-Step” Approach
- Cure SMA-Funded Researcher Publishes Paper on Biomarker Study
- Cure SMA Testifies Before FDA at PDUFA Meeting
- Cure SMA Receives Generous Donation from Fighting for Kaiden Foundation
- Biogen and Ionis Provide Update on Nusinersen (IONIS-SMNRx)
- Abstract Submission Opens for 2016 SMA Researcher Meeting
- Roche Announces New Clinical Trial of RG7916
- AveXis Announces the Completion of Enrollment for Phase 1 Gene Therapy Trial
- Cytokinetics Opens Enrollment for Phase 2 Trial
- Cure SMA Funding Leads to 18 New Research Publications in 2015
- Cure SMA Funds 28 Research Projects in 2015, as Drug Pipeline Grows to 18 Programs
- Cure SMA Reaches Thousands of Families in 2015
- 5th Annual Congressional Dinner Brings Together Families, Government and Industry
- Cure SMA and Cytokinetics Announce Partnership to Advance Education and Awareness of Spinal Muscular Atrophy
- Cure SMA Scientific Advisory Board Meets in Chicago
- Great Progress, Greater Need
- 5th Annual South Florida Gala of Hope Raises Record Total of $224,000
- 5th Annual Hope on the Hill Congressional Dinner Advances SMA Advocacy
- Clinical and Regulatory Webinar and Extended Q&A Now Available
- Fall 2015 Issue of Compass Highlights SMA Researcher Meeting
- Cure SMA Medical Advisory Council Meets in Chicago
- Tell Your Senator to Support Innovations for Healthier Americans
- Cure SMA Welcomes the North Jersey Chapter
- Cure SMA and Other Venture Philanthropy Leaders Share Insight at Partnering for Cures Panel
- Cytokinetics Announces New Details on Upcoming Phase 2 Trial
- Cure SMA Publishes Journal Article on the Patient Voice
- Cure SMA-Funded Researchers Publish Paper in Journal Pediatrics
- Isis Pharmaceuticals Announces SHINE, an Open-Label Extension Study
- Cure SMA to Host Clinical and Regulatory Webinar
- PTC Announces New Clinical Trial Results at World Muscle Congress
- Community Update on ISIS-SMNRx from Biogen and Isis
- New Gene Therapy Results Announced at World Muscle Congress
- Registration Open for the 2016 SMA Researcher Meeting
- House of Representatives Passes Ensuring Access to Clinical Trials Act
- Thank You to Jessica Moyer and Family
- FDA Engagement Update: The Voices of SMA
- 2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 2
- 2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 1
- SMART Gives $50,000 to Cure SMA to Support Research
- Cure SMA and Isis Announce 2015 Holiday Card Contest
- Family Registration for the 2016 Annual SMA Conference is Now Open
- Western New York Teen Wins Nickelodeon Halo Award
- New Update on Clinical Development of RG7800
- 2015 SMA Researcher Meeting Summary: SMA Pathology
- SMA Awareness Month Breakthroughs
- 2015 SMA Researcher Meeting Summary: Drug Discovery
- 2015 SMA Researcher Meeting Summary: Clinical Research
- 2015 SMA Researcher Meeting Summary: Newborn Screening
- Updates from Drug Programs in Clinical Trials
- 2015 SMA Researcher Meeting Summary: Emerging Trends in Motor Neuron Pathobiology
- House of Representatives Passes 21st Century Cures
- Seven Individuals and Families Recognized for Their Contributions to the Community
- Cytokinetics Releases Update on Phase 1 Trial at Annual SMA Conference
- Isis Announces Additional Trial Results at Annual SMA Conference
- Cure SMA Announces Nearly $2 Million in New Planned Research Funding
- Dr. Mary Schroth Joins Cure SMA as Medical Professional Education Consultant
- Read the Summer 2015 Issue of Compass
- The 2015 Annual SMA Conference is Here!
- 2015 Annual SMA Conference Exhibitors
- Cure SMA Releases New Clinical Trial Care Series Booklet
- Isis Releases Encouraging Results from Phase 2 Study
- SMA Drug Pipeline Grows to 18 Programs
- Spring 2015 Directions Posted Online
- Biogen Announces EMBRACE, a New Phase 2 Clinical Trial
- 2015 Annual SMA Conference Booklet Now Posted
- High School Students Lead the Way at Concert for a Cure
- Novartis Launches Phase 2 Clinical Trial
- Cure SMA Announces Partnership with The District Policy Group
- Cure SMA Receives NIH Grant
- In Memory of Ken Emerson
- 21st Century Cures Unanimously Passes Committee
- Collaborative Ways Cure SMA is Driving Research Results
- Meet the SMA Family that Helped Inspire 21st Century Cures
- Cytokinetics and Astellas Release Further Details on Upcoming Phase 2 Trial
- Cure SMA Joins The Research Acceleration and Innovation Network
- Update on SMA-FDA Interactions
- 2015 Family Friendly Poster Session Presenters Announced
- Update on Clinical Development of RG7800
- Cure SMA Funds $1.85 Million in New Research Grants in 2014-2015
- Cure SMA Announces Additional $445,000 for Nationwide Children’s Hospital Gene Therapy Program led by Brian Kaspar, PhD
- New 21st Century Cures Discussion Draft Released
- Registration for the 2015 Annual SMA Conference Closes in Two Weeks
- Cure SMA Publishes New Care Series Booklet on Musculoskeletal System
- Spring 2015 Issue of Compass Now Available
- Annual SMA Conference to Feature Panel of Leading SMA Drug Experts
- Muscles for Mckenna Gala Raises Over $100,000
- Cure SMA Welcomes the Virginia Chapter
- Cure SMA Releases the Schedule for the 2015 Annual SMA Conference
- Cure SMA Announces New $140,000 Basic Research Grant to Chad Heatwole, MD, at the University of Rochester
- SMA Researcher Meeting to Host Over 100 Presentations
- Biogen Announces NURTURE, a New Phase 2 Clinical Trial
- Cure SMA Announces Additional $315,000 for SMA Drug Program at The California Institute of Biomedical Research
- Cure SMA Releases Updated Overview of Research Model
- Cure SMA Hosts SMA Researcher Meeting this June
- How Our Cure SMA Community is Raising Awareness
- Updates on Pharmaceutical Companies Investing in SMA Drug Development
- Cure SMA Awards $140,000 Grant to Francesco Lotti, PhD, of Columbia University
- 21st Century Cures Discussion Document Released
- Cure SMA-Funded Researcher Publishes Paper on Critical Issues for SMA Families and Doctors
- Join Cure SMA in Celebrating the International Day of Acceptance
- Roche Buys Trophos to Continue Work on SMA Drug
- Cure SMA Announces New $30,000 Clinical Care Research Grant to Deborah Boroughs at BAYADA Home Health Care
- Cure SMA Awards $140,000 Grant to Sara Custer, PhD, of Indiana University
- Together, We Made History
- Cure SMA Announces New $50,000 Care Research Grant to Rebecca Davis in the Lab of Dr. Swoboda at University of Utah
- NIH Releases Summary of the SO-SMART Workshop
- Cytokinetics and Astellas Announce New Phase 2 Clinical Trial
- Cure SMA Announces New $50,000 Clinical Care Research Grant to Nilesh Mehta at Boston Children’s Hospital
- Applications Open for 2015 Annual SMA Conference Scholarships
- Cure SMA Provides Record Number of Resources to Families Worldwide
- Cure SMA Funding Leads to 16 New Research Publications in 2014
- Hope on the Hill Advances Key SMA Legislative Priorities
- Cure SMA Funds 26 Research Projects in 2014
- Isis Expands Phase 3 Clinical Trials
- Cure SMA Researchers Present at Society for Neuroscience Meeting
- PTC Therapeutics Announces New Phase 1a/2b Clinical Trial
- Isis Announces Second Phase III Clinical Trial
- Southern California Gala of Hope Brings Community and Industry Partners Together to Raise $80,000
- Cure SMA Scientific Advisory Board Meets in Washington D.C.
- Families, Government, and Industry Leaders Unite to Help Cure SMA
- Together, We Can Cure SMA
- Promising Results Published on Cure SMA-Funded Gene Therapy Project
- Read the Fall 2014 Issue of Compass
- Cure SMA Honors Scott Geller with 1st Annual Community Partnership Award
- Isis Releases Phase II Clinical Trial Results
- AveXis Announces the Completion of Dosing of the Low-Dose Cohort in US Clinical Trial for Spinal Muscular Atrophy
- AveXis Receives Orphan Drug Designation
- Isis Begins Recruiting for Phase III Trial
- Cure SMA-Funded CALIBR Research Collaboration Expanded to USC
- Cure SMA and Isis Announce Holiday Card Contest TEST
- Cure SMA and Isis Announce Holiday Card Contest
- Cure SMA and PPMD Invite Spinal Muscular Atrophy Parents to Take Part in Two Clinical Trial Surveys
- Cure SMA Featured in Assay and Drug Development Technologies
Industry Updates
- Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate
- New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA
- Scholar Rock Announces Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA)
- Biogen Announces Positive Topline Results from Study of Higher Dose Regimen of Nusinersen, Showing Significant Benefit in Treatment of SMA
- SMA Highlights: Updates from Biogen
- Five-Year Data for Genentech’s Evrysdi Show the Majority of Treated Children With a Severe Form of SMA Achieved or Maintained the Ability to Sit, Stand or Walk
- Novartis Gene Therapies Releases Spring SMA Community Letter
- Genentech Releases SMA Community Letter Recapping Spring Activities
- Biogen Releases SMA Community Letter
- Apitegromab Linked to Improved Outcomes for Those with Spinal Muscular Atrophy
- New Biomarker Data Add Further Evidence Supporting the Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs after Gene Therapy
- Novartis Presents New Data on Safety and Efficacy of Zolgensma, Including Maintained and Improved Motor Milestones in Older and Heavier Children with SMA
- Novartis Gene Therapies Releases SMA Community Statement
- Genentech Releases SMA Community Letter with Evrysdi (risdiplam) Updates
- Biogen Shares DEVOTE and RESPOND Study Updates
- Genentech Shares 1-Year Primary Analysis from RAINBOWFISH
- Novartis Gene Therapies SMA Community Update
- SMA Highlights and Updates from Biogen
- NMD Pharma Initiates Phase II Trial for SMA
- Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial
- Genentech Releases SMA Community Letter
- Cure SMA Announces Expanded Phase 3 of Real World Evidence Collaboration
- Community Update from Genentech
- Novartis Gene Therapies Release May 2023 SMA Community Update
- Biogen Releases Q1 2023 Community Statement
- Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset
- Biohaven’s Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy
- Genentech Releases SMA Community Letter
- Biogen Releases Q3 SMA Community Statement
- Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
- New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA
- SMA Community Update from Novartis Gene Therapies
- SMA Community Update from Novartis Gene Therapies
- Community Statement from Novartis Gene Therapies: Zolgensma Safety Monitoring Update on Acute Liver Failure
- Biogen Shares Community Statement for SMA Awareness Month
- SMA Community Update from Novartis Gene Therapies
- FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy
- Biohaven Licenses a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA)
- Biogen Exercises Option with Ionis to Develop and Commercialize Investigational ASO for SMA
- Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With SMA
- Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
- Community Update from Novartis Gene Therapies
- Update on Genentech/Roche Initiation of MANATEE Clinical Study
- New Data Presented at World Muscle Society 2021 Virtual Congress
- Community Statement from Biogen: New Clinical Study Planned to Evaluate the Potential Benefit of an Investigational Higher Dose of Nusinersen in People with SMA Previously Treated with Evrysdi® (risdiplam)
- Novartis Announces Lift of Partial Clinical Trial Hold, Plans to Initiate a New Study of Intrathecal Zolgensma in Older Patients with SMA
- Data for Genentech’s Evrysdi Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies with Type 1 SMA
- Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children
- Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA
- New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA
- Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference
- Community Statement: Quarterly Update from Novartis Gene Therapies
- Community Statement from Novartis Gene Therapies
- Genentech Shares New Evrysdi Data on Improved Motor Function and Survival in Babies with SMA Type 1
- Biogen Shares New Spinraza Data Exploring Opportunities to Improve Outcomes for SMA Patients
- Scholar Rock Community Statement on TOPAZ 12-Month Topline Data
- Community Statement from Genentech
- Community Statement from Novartis Gene Therapies
Latest News
- Make an Impact with Cure SMA’s Mission, Milestones, Momentum Campaign
- Check Out Cure SMA’s 2023 State of SMA Report
- Genentech Shares 1-Year Primary Analysis from RAINBOWFISH
- Key Congressional Committee Urges NIH to Address Unmet Needs of Individuals with SMA
- New DOT Rule Will Require Truly Accessible Lavatories on New Single Aisle Aircraft
- SMA Scientists and Clinicians from Around the World Gather at the 27th Annual SMA Research & Clinical Care Meeting
- Big Week in Washington for Cure SMA-Supported Legislation
- Cure SMA’s 2022 State of SMA Report
- Individuals with SMA Describe New Travel Hiccups and Wheelchair Damage in Their Air Travels to Cure SMA National Conference
- Luke 18:1 Foundation Donates $500,000 to Cure SMA to Support Groundbreaking SMA Research and Care
- Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA
- Community Update from Genentech
- Congressional Committee Approves Legislation to Improve Air Travel for People with SMA and other Disabilities
- Erin Trainor Memorial Fund will Match Donations for the funding of Care Centers
- Cure SMA Applauds Federal Agency Decision that Should Lead to Better Insurance Coverage for Seat Elevation Systems in Power Wheelchairs
- Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset
- Cure SMA Awards $100,000 Grant to Saravanan Arumugam, PhD, Universidad de Seville
- Cure SMA Holds Patient-Led Listening Session with FDA
- A Deliciously Easy Way to Fundraise for Cure SMA
- Special NBS Awareness Month Announcement: South Carolina Screens for SMA!
- SMA Community Update from Novartis Gene Therapies
Our Impact
- SMA Community Advocacy Wins and Champions Celebrated at Cure SMA Event
- Celebrate 40 Years of Impact – Join Us in Making a Difference!
- Make an Impact with Cure SMA’s Mission, Milestones, Momentum Campaign
- Congressional Action Recognizes Unmet Needs of SMA Community
- Spring 2024 Issue of Compass Now Available
- Cure SMA Announces Additional $750,000 in Basic Research Funding
- Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Industry Collaboration Initiatives
- Cure SMA Awards $150,000 Grant to Lyndsay Murray,PhD, at the University of Edinburgh in Scotland
- Government Funding Bill Supports New SMA Research to Address Unmet Needs
- Cure SMA Awards $114,000 Grant to Michael Tellier, PhD, at the University of Leicester in the United Kingdom
- Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, at The Medical College of Wisconsin
- Cure SMA Awards $100,000 Grant to Elana Molotsky, PhD, at the Johns Hopkins University School of Medicine
- Cure SMA Awards $150,000 Grant to Melissa Bowerman, PhD, at Keele University
- 100% of States Now Screening Newborns for SMA
- Nevada Starts Screening for SMA – Only One State Remains!
- Analysis: Cure SMA’s Risk/Benefit Survey
- Administration Officials & Members of Congress Highlight SMA Community Advocacy at 12th Annual Hope on the Hill Washington, D.C. Event
- August is SMA Awareness Month!
- Cure SMA Awards $100,000 Grant to Saravanan Arumugam, PhD, Universidad de Seville
- District of Columbia Starts Screening for SMA During Rare Disease Month
- Cure SMA Awards $150,000 Grant to Florence Rage, PhD, at the Institute of Molecular Genetics of Montpellier
- Cure SMA Awards $150,000 Grant to Anton Blatnik, PhD, at The Ohio State University
- Cure SMA to Host 11th Annual Hope on the Hill Event in Washington, D.C.
- The Giving Season is Here!
- SMA Community Seeks SSI Improvements on Program’s 50th Anniversary
- Cure SMA Holds Patient-Led Listening Session with FDA
- Cure SMA Holds Patient-Led Listening Session with FDA
- Special NBS Awareness Month Announcement: South Carolina Screens for SMA!
- Special NBS Awareness Month Announcement: South Carolina Screens for SMA!
- Seat Elevators on Power Wheelchairs are a Necessity, not a Luxury, according to the SMA Community
- Cure SMA Launches Benefit-Risk Survey to Assess Patient Perspective on Treatment Possibilities and Risks
- Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships
- Cure SMA Announces Expanded Phase 7 of SMA Industry Collaboration
- Cure SMA Releases Updated Drug Pipeline
- Survey Alert: SMA Family Caregivers
- In Just Three Years, Cure SMA Marks Nationwide Milestone for 85% of U.S. Babies Now Screened at Birth for SMA
- Cure SMA Publishes Manuscript on SMA Awareness, Screening, and Referral Patterns Amongst Pediatricians
- Cure SMA’s New Year Letter for 2021
- Cure SMA, Novartis Publish Economic Burden of SMA Analysis in Journal of Market Access & Health Policy
- Results from Annual SMA Community Update Survey Now Published
- Annual Cure SMA Community Update Survey Ready for Your Input
- Cure SMA Launches “Spotlight on SMA” Partnership with Neurology Reviews
- Cure SMA Establishes an Adult Advisory Council
- Cure SMA Awards Grants to Four States to Expediate the Implementation of SMA Newborn Screening
- Updated SMA Drug Pipeline Now Available
- Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado
- Spring 2019 SMA Newborn Screening Update
- Community Update Survey Q&A
- Cure SMA Care Center Network Expands to 10 Sites
- Cure SMA Awards $200,000 Grant to Charlotte Sumner, MD, Johns Hopkins University
- Cure SMA and Parent Project Muscular Dystrophy Announce Collaboration
- Cure SMA Awards $150,000 Grant to Veronica Pessino, PhD, Salk Institute for Biological Sciences
- Cure SMA Awards $150,000 Grant to Laxman Gangwani, PhD, Texas Tech University
- Cure SMA Announces Expanded Phase 3 of SMA Industry Collaboration
- Hope on the Hill Congressional Dinner Raises $200,000
- 2018 Research Year-in-Review
- Cure SMA Funding Leads to 16 New Research Publications in 2018
- Advocacy Year-in-Review 2018
- Fall 2018 Issue of Compass Now Available
- Cure SMA Launches “SMArt Moves” for Early Diagnosis and Treatment
- Kansas Adopts SMA Newborn Screening as Several Other States Move Closer to Adoption
- North Carolina to Screen Newborns for SMA Through Early Check
- SMA Newborn Screening Advancements
- New York to Implement Newborn Screening for SMA October 1st
- Cure SMA Provides Funding for “Patient Reported Impact of Symptoms in Spinal Muscular Atrophy (PRISM-SMA)” Published in Neurology
- Illinois Adopts Permanent SMA Screening
- Community Spotlight: Brynne Willis
- Wisconsin Moves One Step Closer to SMA Newborn Screening
- Cure SMA Awards $300,000 Grant to Kevin Hodgetts, PhD, The Brigham and Women’s Hospital
- Cure SMA Awards $300,000 Grant to Umrao Monani, PhD, Columbia University
- HHS Secretary, Alex Azar, Recommends Nationwide Newborn Screening for SMA
- SMA Newborn Screening Moves Forward in New Jersey
- Summer 2018 Issue of Compass Now Available
- Ohio Moves One Step Closer to SMA Newborn Screening
- Cure SMA Awards $150,000 Grant to Kathryn Swoboda, MD, Massachusetts General Hospital
- Spring 2018 Issue of Directions Now Available
- Indiana Becomes Fourth State to Adopt Newborn Screening for Spinal Muscular Atrophy
- SMA Advocates Meet With House and Senate Offices
- Cure SMA Awards $150,000 Grant to Chad Heatwole, MD, University of Rochester
- The Discovery of Spinraza
- There is Still Time to Complete Cure SMA’s 2018 Community Survey
- Cure SMA Awards $150,000 Grant to Bakri Elsheikh, MD, Ohio State University Wexner Medical Center
- Cure SMA Database Findings Published in the Journal of Neuromuscular Diseases
- SMA Newborn Screening Guidelines Published in Journal of Neuromuscular Diseases
- Cure SMA Welcomes Back AveXis as a 2018 National Premier Partner
- Cure SMA Awards $150,000 Grant to Brunhilde Wirth, PhD, Institute of Cologne, Germany
- Cure SMA Awards $50,000 Grant to Rashmi Kothary, PhD, University of Ottawa
- Cure SMA Awards $150,000 Grant to Robin Parks, PhD, University of Ottawa
- Cure SMA Awards $75,000 Grant to Robert Kalb, MD, Northwestern University
- Cure SMA Awards $150,000 Grant to Umrao Monani, PhD, Columbia University
- Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, Medical College of Wisconsin
- Awareness is Best Raised When We Work Together
- Additional Funding for SMA Care Centers Announced
- Advisory Committee on Heritable Disorders in Newborns and Children Recommends Nationwide Newborn Screening for Spinal Muscular Atrophy
- Utah Becomes First State to Implement Permanent Screening for SMA
- Federal Committee to Vote on Spinal Muscular Atrophy Newborn Screening on Thursday
- SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report
- Cure SMA Launches Advocacy Action Network
- Minnesota Becomes Second State to Adopt Newborn Screening for SMA
- Biogen and Ionis Enter Into New Collaboration to Identify Novel Therapies for the Treatment of Spinal Muscular Atrophy
- Cure SMA Assists Hundreds of Families in 2017
- 2017 Research Year-in-Review
- Cure SMA-Funded Research Results in 21 Published Journal Articles
- Cure SMA-Funded Researcher, Stephen Kolb, Publishes Paper
- 7th Annual Hope on the Hill Congressional Dinner Achieves $1 Million Milestone
- SMA Advocates Testify at Federal Newborn Screening Meeting
- Fall 2017 Issue of Compass Now Available
- Cure SMA Launches Benefit-Risk Survey to Address Important Issues in Treatment Access and Future Drug Approvals in SMA
- Cure SMA Meets in Utah with State and Federal Elected Officials to Support Newborn Screening
- Cure SMA Contributes Article to a Special Issue of the Journal Gene Therapy
- Centers for Medicare and Medicaid Services (CMS) Survey
- SMA Families and Cure SMA Staff Visit Capitol Hill for Advocacy Efforts
- RUSP Nomination for SMA Accepted into Evidence Review
- SMA Community’s Voice Heard “Loud and Clear” at Last Week’s Patient Focused Drug Development Meeting with the FDA
- Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University
- SMA is Submitted to the Federal Newborn Recommended Uniform Screening Panel (RUSP)
- Registration Opens for the SMA Patient-Focused Drug Development Meeting with the FDA
- Cure SMA Assists Hundreds of Families in 2016
- Cure SMA Funding Results in 16 New Research Publications
- FDA Approves Spinraza for SMA
- Cure SMA Participates in a Special FDA Listening Session
- Cure SMA Announces Additional Conference Support for Newly Diagnosed Families
- Help Us Shape the Future Together, and Reach our Goal of $750,000
- Over 35 Cure SMA-Funded Researchers Contribute to Comprehensive SMA Encyclopedia
- Cure SMA Launches Insurance Coverage and Payment Policy Project
- Cure SMA to Host Webinar on the New Drug Application Process
- 6th Annual Congressional Dinner Furthers SMA Advocacy Work
- SMA Community is Granted a Patient Focused Drug Development Meeting with the FDA
- Cure SMA Announces Newborn Screening Initiative in Partnership with MDA
- Summer 2016 Issue of Compass Now Available
- We’re Partnering With The Mighty!
- Cure SMA Plans a Series of Educational Modules for the SMA Community
- Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA
- Cure SMA Announces $2.5 Million in New Planned Research Funding
- Cure SMA Posts Keynote Presentations on the NDA Process
- Cure SMA Funds $2 Million in New Research in 2015-2016
- The 2016 Annual SMA Conference is Here!
- Cure SMA Receives 2016 NIH Grant
- Cure SMA Releases June 2016 Update to the SMA Drug Pipeline
- 2016 Update on SMA-FDA Interactions
- Spring 2016 Issue of Compass Now Available
- Cure SMA Awards $70,000 Grant to Christine DiDonato, Lurie Children’s Hospital of Chicago
- Cure SMA-Funded Researchers Receive NIH Grant
- Cure SMA Awards $30,000 Grant to Jocelyn Cote, PhD, University of Ottawa
- Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University
- Cure SMA Awards $90,000 Grant to Antoine Cl̩ry, PhD, ETH Zurich
- Cure SMA Awards $140,000 Grant to Christine Beattie, PhD, Ohio State University
- Cure SMA-Funded Researchers Publish Paper on Motor Neuron and Astrocyte Defects
- Cure SMA Awards $140,000 Grant to Rashmi Kothary, MD, Ottawa Hospital Research Institute
- Cure SMA Awards $140,000 Grant to Megerditch Kiledjian, PhD, of Rutgers
- Cure SMA Testifies Before FDA at PDUFA Meeting
- Cure SMA Funding Leads to 18 New Research Publications in 2015
- Cure SMA Funds 28 Research Projects in 2015, as Drug Pipeline Grows to 18 Programs
- Cure SMA Reaches Thousands of Families in 2015
- Great Progress, Greater Need
- Cure SMA and Other Venture Philanthropy Leaders Share Insight at Partnering for Cures Panel
- Cure SMA-Funded Researchers Publish Paper in Journal Pediatrics
- Cure SMA Announces Nearly $2 Million in New Planned Research Funding
- SMA Drug Pipeline Grows to 18 Programs
- Collaborative Ways Cure SMA is Driving Research Results
- Update on SMA-FDA Interactions
- Cure SMA Funds $1.85 Million in New Research Grants in 2014-2015
- Cure SMA Announces Additional $445,000 for Nationwide Children’s Hospital Gene Therapy Program led by Brian Kaspar, PhD
- Spring 2015 Issue of Compass Now Available
- Cure SMA Announces New $140,000 Basic Research Grant to Chad Heatwole, MD, at the University of Rochester
- Cure SMA Announces Additional $315,000 for SMA Drug Program at The California Institute of Biomedical Research
- Cure SMA Awards $140,000 Grant to Francesco Lotti, PhD, of Columbia University
- Cure SMA Awards $140,000 Grant to Sara Custer, PhD, of Indiana University
- Together, We Made History
- NIH Releases Summary of the SO-SMART Workshop
- Help Us Make History with $750,000 for SMA Research
- Cure SMA Provides Record Number of Resources to Families Worldwide
- Cure SMA Funding Leads to 16 New Research Publications in 2014
- Cure SMA Funds 26 Research Projects in 2014
- SMA Drug Pipeline Continues to Grow
- Cure SMA Scientific Advisory Board Meets in Washington D.C.
- Families, Government, and Industry Leaders Unite to Help Cure SMA
- Medical Advisory Council Gathers for Fall 2014 Meetings
- Promising Results Published on Cure SMA-Funded Gene Therapy Project
- Read the Fall 2014 Issue of Compass
- Cure SMA-Funded CALIBR Research Collaboration Expanded to USC
- Cure SMA Announces Basic Research Funding for Dr. Lyndsay Murray
- Cure SMA Featured in Assay and Drug Development Technologies
- Cure SMA Reaches Out to Newly Diagnosed Families
- Cure SMA Announces New Research Funding
Research
- Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate
- Scholar Rock Announces Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA)
- Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Real World Evidence Collaboration Initiatives
- Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships
- Cure SMA Publishes Update on the Birth Prevalence of Spinal Muscular Atrophy (SMA)
- Special Session Highlights from 2024 Annual SMA Research & Clinical Care Meeting
- Clinical Research and Drug Development Session Highlights from 2024 Annual SMA Research & Clinical Care Meeting
- Spring 2024 Issue of Compass Now Available
- SMA Researchers and Clinicians from Around the World Gather at the 28th Annual SMA Research & Clinical Care Meeting
- Check Out Cure SMA’s 2023 State of SMA Report
- Cure SMA Announces Additional $750,000 in Basic Research Funding
- Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Industry Collaboration Initiatives
- Cure SMA Launches New Resources to Enhance Clinical Trial Access
- Cure SMA Announces Expanded Phase 9 of SMA Industry Collaboration
- Participate in the Annual Cure SMA Community Update Survey – Open Now!
- Cure SMA Awards $150,000 Grant to Lyndsay Murray,PhD, at the University of Edinburgh in Scotland
- Cure SMA Awards $114,000 Grant to Michael Tellier, PhD, at the University of Leicester in the United Kingdom
- Apitegromab Linked to Improved Outcomes for Those with Spinal Muscular Atrophy
- Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, at The Medical College of Wisconsin
- Cure SMA Awards $100,000 Grant to Elana Molotsky, PhD, at the Johns Hopkins University School of Medicine
- Cure SMA Awards $150,000 Grant to Melissa Bowerman, PhD, at Keele University
- Analysis: Cure SMA’s Risk/Benefit Survey
- Identifying Biomarkers of Spinal Muscular Atrophy for Further Development
- Biohaven Completes Enrollment in Pivotal Phase 3 Study
- Clinical Research and Drug Development Session Highlights from 2023 SMA Research & Clinical Care Meeting
- Cure SMA Announces Expanded Phase 3 of Real World Evidence Collaboration
- SMA Scientists and Clinicians from Around the World Gather at the 27th Annual SMA Research & Clinical Care Meeting
- Cure SMA’s 2022 State of SMA Report
- Cure SMA Awards $100,000 Grant to Saravanan Arumugam, PhD, Universidad de Seville
- Cure SMA Awards $75,000 Grant to Lyndsay Murray, PhD, University of Edinburgh
- Biohaven’s Taldefgrobep Alfa Receives FDA Fast Track Designation for Spinal Muscular Atrophy
- Cure SMA Awards $150,000 Grant to Florence Rage, PhD, at the Institute of Molecular Genetics of Montpellier
- Cure SMA Awards $150,000 Grant to Anton Blatnik, PhD, at The Ohio State University
- Cure SMA Supports Clinical Trial Readiness
- Cure SMA Awards $150,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires
- Cure SMA Holds Patient-Led Listening Session with FDA
- Cure SMA Holds Patient-Led Listening Session with FDA
- Cure SMA Launches Benefit-Risk Survey to Assess Patient Perspective on Treatment Possibilities and Risks
- 2022 Cure SMA Annual Research Meeting Summaries – Special and Clinical Research Session
- 2022 Cure SMA Annual Research Meeting Summaries – Basic Research Sessions
- Cure SMA Advocacy Campaign Leads to Congressional Support for More SMA Research
- Cure SMA to Hold Patient-Led Listening Session with FDA
- Cure SMA Attends Spring 2022 Conferences to Promote Findings from Industry Collaboration Initiatives
- FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy
- Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships
- Cure SMA Announces Expanded Phase 7 of SMA Industry Collaboration
- Biohaven Licenses a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA)
- International Day of Women and Girls in Science
- Biogen Exercises Option with Ionis to Develop and Commercialize Investigational ASO for SMA
- Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With SMA
- Cure SMA Releases Updated Drug Pipeline
- Survey Alert: SMA Family Caregivers
- Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
- Cure SMA Discusses FDA-Approved SMA Treatments & Dissects Combination Therapy
- New Data Presented at World Muscle Society 2021 Virtual Congress
- Data for Genentech’s Evrysdi Published in New England Journal of Medicine Shows Significant Improvement in Survival and Motor Milestones in Babies with Type 1 SMA
- Cure SMA Convenes Working Group to Discuss Biomarkers for SMA
- Novartis Data Demonstrate Age-Appropriate Development When Zolgensma Is Used Presymptomatically and Rapid, Clinically Meaningful Efficacy in Symptomatic Children
- Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 SMA
- New Genentech Data for Evrysdi Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People with SMA
- Biogen Presents New Spinraza Data at the 2021 Virtual SMA Conference
- Cure SMA Publishes Manuscript on SMA Awareness, Screening, and Referral Patterns Amongst Pediatricians
- Cure SMA-Funded Researchers at Ohio State University Publish Paper on Effect of Nusinersen in Adults with SMA
- Cure SMA-Funded Researchers at Columbia University Publish Paper on Fatigue in SMA
- Cure SMA Attends Spring 2021 Professional Organization Conferences to Promote Findings from Industry Collaboration Initiatives
- Genentech Shares New Evrysdi Data on Improved Motor Function and Survival in Babies with SMA Type 1
- Biogen Shares New Spinraza Data Exploring Opportunities to Improve Outcomes for SMA Patients
- Participate in Annual Cure SMA Community Update Survey – Open Now!
- Cure SMA Presents Data on Cure SMA Care Center Network and SMA Clinical Data Registry
- Scholar Rock Community Statement on TOPAZ 12-Month Topline Data
- Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on SMA-HI, a Clinical Trial Outcome in Adults with SMA
- Nusinersen Research Updates Presented by Biogen
- Research Updates from Scholar Rock
- Evrysdi Research Updates from Genentech
- Zolgensma Research Updates from Novartis Gene Therapies
- Cure SMA Publishes Quality of Life in Teens and Young Adults Manuscript in Orphanet Journal of Rare Diseases
- Results from Genentech’s Evrysdi Study in Infants with Type 1 SMA Published in NEJM
- Cure SMA Offer Perspectives on Combination Therapy for SMA
- Cure SMA Funds Over $9 Million in New and Ongoing Research in FY20
- Cure SMA, Novartis Publish Economic Burden of SMA Analysis in Journal of Market Access & Health Policy
- Biogen SMA Q4 2020 Community Statement
- Results from Annual SMA Community Update Survey Now Published
- Scholar Rock Issues Community Statement with Update on SRK-015 in Patients with Types 2 and 3 SMA
- NeurologyLive Article Now Available, Featuring an Interview with Cure SMA’s Dr. Mary Schroth
- Cure SMA Publishes Paper in Partnership with NeurologyLive on Impact of COVID-19
- Novartis Presents New Zolgensma® Data Further Demonstrating Therapeutic Benefit
- Biogen Q3 2020 Community Statement: COVID-19 Updates and Latest Milestones
- Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of SMA
- Genentech Releases Evrysdi™ FDA Approval Letter to the SMA Community
- Genentech Receives FDA Approval of Evrysdi (risdiplam) for the Treatment of SMA
- Genentech Announces 2-Year Risdiplam Data From SUNFISH and New Data From JEWELFISH in Infants, Children and Adults with SMA
- Biogen Shares Results from Landmark NURTURE Study of Pre-Symptomatic SMA Patients Treated with Spinraza
- Cure SMA to Fund Network of Prominent SMA Clinical Research and Treatment Centers
- Biogen Shares SPINRAZA Research Update
- Biogen Releases Q2 Community Statement on Latest Milestones in Clinical Development Program
- Update from Scholar Rock on TOPAZ Phase 2 Clinical Trial of SRK-015 in Patients with SMA
- Genentech’s Risdiplam Shows Significant Improvement in Survival and Motor Milestones in Infants With Type 1 SMA
- Annual Cure SMA Community Update Survey Ready for Your Input
- Grant Announcement: Cure SMA Awards $190,000 Grant to Stephen Meriney, Ph.D., University of Pittsburgh
- Biogen Announces First Patient Treated With Higher Dose Of Spinraza In Phase 2/3 DEVOTE Study
- Zolgensma® Data Shows Rapid, Significant, Clinically Meaningful Benefit in SMA, Including Prolonged Event-free Survival, Motor Milestone Achievement and Durability, Now Up to 5 Years Post-dosing
- Cure SMA Launches Part 2 of “Spotlight on SMA” in Partnership with Neurology Reviews
- Grant Announcement: Cure SMA Awards $150,000 Grant to Emily Welby, Ph.D., Medical College of Wisconsin
- Biogen Issues Community Letter with Perspective on Coronavirus (COVID-19)
- Grant Announcement: Cure SMA Awards $190,000 Grant to Umrao Monani, Ph.D., Columbia University
- Cure SMA Awards $100,000 Grant to Rashmi Kothary, Ph.D., Ottawa Hospital Research Institute
- Grant Announcement: Cure SMA Awards $150,000 Grant to Timra Gilson, Ph.D., Indiana University
- Grant Announcement: Cure SMA Awards $150,000 Grant to Dmytro Morderer, Ph.D., Mayo Clinic, Jacksonville, FL
- Grant Announcement: Cure SMA Awards a $170,000 Grant to Lyndsay Murray, Ph.D., University of Edinburgh
- Cure SMA Working Group Revises Recommendations for the Treatment of Infants Diagnosed with SMA via Newborn Screening Who Have 4 Copies of SMN2
- Cure SMA Launches “Spotlight on SMA” Partnership with Neurology Reviews
- Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 Years with Type 2 or Type 3 SMA
- Genentech’s Risdiplam Meets Primary Endpoint in Pivotal FIREFISH Trial in Infants with Type 1 SMA
- Cure SMA and Biogen to Co-Host Webinar on DEVOTE Clinical Trial – Register Today!
- A Look Back at 2019: Cure SMA Research Year-in-Review
- Biogen Issues Q4 2019 Community Statement on Spinraza
- Fall 2019 Issue of Compass Now Available
- Check Out the Cure SMA Booklet on “Scientific Considerations for Drug Combinations”
- SMA Concordance Among Siblings Published in the Journal of Neuromuscular Diseases
- Scholar Rock Reports Preliminary Pharmacokinetic and Pharmacodynamic Data from TOPAZ Phase 2 Trial of SRK-015 for the Treatment of Patients with Spinal Muscular Atrophy
- Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SUNFISH Trial in People With Type 2 or 3 Spinal Muscular Atrophy
- AveXis Issues Community Statement on AVXS-101 Clinical Hold
- Novartis Announces AVXS-101 Intrathecal Study Update
- AveXis Presents Updated STRONG Data Demonstrating a Higher Mean Increase in Hammersmith Functional Motor Scale-Expanded (HFMSE) Scores Among Older SMA Type 2 Patients Following One-time Intrathecal Administration of AVXS-101
- Scholar Rock to Present Preclinical and Phase 1 Clinical Data on SRK-015 at the World Muscle Society Congress
- Landmark NURTURE Study of Infants with Spinal Muscular Atrophy (SMA) Treated Pre-Symptomatically with Spinraza Published in Neuromuscular Disorders
- Risdiplam Data Presented at World Muscle Society Congress Demonstrates Continued Benefit for Patients with Spinal Muscular Atrophy
- AveXis Presents New Data at EPNS Continuing to Show Significant Therapeutic Benefit of Zolgensma® in Prolonging Event-free Survival Now up to 5 Years of Age in Patients with SMA Type 1
- Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study, DEVOTE, Evaluating a Higher Dose of SPINRAZA and Additional Data in a Broad Range of Patients
- Zolgensma Update and Approval Webinars Now Available Online
- 2019 Researcher Meeting Summary: Clinical Research Studies for SMA
- 2019 SMA Researcher Meeting Summary: Basic Research Podium Presentations
- 2019 SMA Researcher Meeting Summary: Special Session on Spinal and Neuromuscular Circuitry: Exploring Defects in SMA and Potential Therapeutic Targets
- Biogen Announces New NURTURE Data at the 2019 Annual SMA Conference
- Updated SMA Drug Pipeline Now Available
- Cytokinetics Releases New Data from Reldesemtiv Clinical Study at the 2019 Annual SMA Conference
- Family Friendly Research Poster Session Presenters Announced
- Biogen Issues Q2 Community Statement on Spinraza
- Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers
- AveXis and Cure SMA to Co-Host Webinar on the FDA’s Approval of Zolgensma
- AveXis Issues Community Statement on Zolgensma Approval
- AveXis Receives FDA Approval of Zolgensma, a Gene Therapy, for Spinal Muscular Atrophy for Patients Under Two Years of Age
- Cure SMA Awards $150,000 Grant to Krysta Engel, PhD, University of Colorado
- Novartis Issues Community Statement on Branaplam Clinical Study
- Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA
- Dr. Arthur Burghes Named 2019 Distinguished Scholar
- Scholar Rock Issues Community Statement on the Initiation of a New Clinical Trial for SRK-015
- Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in Spinal Muscular Atrophy at the AAN Annual Meeting
- AveXis Presents Data at AAN Annual Meeting Demonstrating Efficacy of Zolgensma in Broad Spectrum of Spinal Muscular Atrophy (SMA) Patients
- Biogen to Present Data at AAN Highlighting Spinraza and Emerging Biomarkers for SMA
- Genentech Presents New Data at the 2019 AAN Annual Meeting
- Biogen Presents New Data Regarding Adults and Infants with SMA
- Community Update Survey Q&A
- Cure SMA Awards $150,000 Grant to Jocelyn Cote, PhD, University of Ottawa
- Cure SMA Awards $200,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires
- Cure SMA Awards $200,000 Grant to Charlotte Sumner, MD, Johns Hopkins University
- The Winter 2019 issue of Compass in Now Available Online
- Record Number of Abstracts Submitted for the 2019 SMA Researcher Meeting
- Cure SMA Launches Third Annual Community Survey to Address Important Issues in SMA Treatment
- Recording of Genentech/Roche and Cure SMA Webinar Now Available Online
- Cure SMA Advocates for SMA Community at ICER Hearing
- Biogen Provides 2019 Community Update on Spinraza
- Genentech/Roche and Cure SMA to Co-Host Webinar
- Scholar Rock Announces Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans
- Cure SMA Awards $150,000 Grant to Veronica Pessino, PhD, Salk Institute for Biological Sciences
- Cure SMA Awards $150,000 Grant to Laxman Gangwani, PhD, Texas Tech University
- Cytokinetics Announces Receipt of FDA Feedback Regarding Reldesemtiv in Patients with SMA
- Genentech, Roche and PTC Therapeutics Provide Update on Regulatory Filing for Risdiplam
- Teen and Young Adult Survey Extended Through January 21st
- Cure SMA Announces Expanded Phase 3 of SMA Industry Collaboration
- December Webinar on SMA Treatments and Clinical Trials Now Available Online
- 2018 Research Year-in-Review
- Cure SMA Funding Leads to 16 New Research Publications in 2018
- Biogen Issues Q4 Community Statement on Spinraza
- AveXis Issues Community Statement on BLA Acceptance
- Fall 2018 Issue of Compass Now Available
- Novartis Announces FDA Filing Acceptance and Priority Review for AVXS-101
- Scholar Rock Announces Publication of Preclinical Data on the Therapeutic Benefit of Inhibiting Myostatin Activation in Models of SMA
- Breakthrough Prize Awarded to Adrian Krainer and Frank Bennett for SMA Research Leading to Spinraza
- New Spinraza Data Presented at Annual Congress of the World Muscle Society Demonstrate Benefits in Treating Presymptomatic Infants with SMA
- Cytokinetics and Astellas Provide Clinical Trial Updates for Reldesemtiv in SMA
- Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress
- Genentech Releases Community Statement on Risdiplam
- Cure SMA’s Approach to Newborn Screening
- Cure SMA Provides Funding for “Patient Reported Impact of Symptoms in Spinal Muscular Atrophy (PRISM-SMA)” Published in Neurology
- Updated 2018 SMA Standards of Care Statements Available Online
- 2018 SMA Researcher Meeting Summary: SMA Therapy Development
- 2018 SMA Researcher Meeting Summary: Clinical Research Studies for SMA
- 2018 SMA Researcher Meeting Summary: Basic Research Podium Presentations
- 2018 SMA Researcher Meeting Special Session Summary: Clinical and Basic Questions of SMA in the Era of SMN Repletion: Challenge and Opportunity
- Cure SMA Awards $300,000 Grant to Kevin Hodgetts, PhD, The Brigham and Women’s Hospital
- Dr. John Kissel, Chair of Neurology at OSU Wexner Medical Center, Retires After 40 Years
- Roche Releases Community Update on Risdiplam
- Cure SMA Awards $300,000 Grant to Umrao Monani, PhD, Columbia University
- Summer 2018 Issue of Compass Now Available
- PTC Therapeutics Announces Risdiplam (RG7916) is Well Tolerated at All Dose Levels With No Drug-Related Safety Findings
- Cytokinetics Presents Data from the Phase 2 Clinical Trial of Reldesemtiv (CK-2127107) in Patients with SMA at the 2018 Annual SMA Conference
- Biogen to Present New Data at the 2018 Annual SMA Conference
- Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015
- Roche Releases Community Statement on Olesoxime Program
- Cure SMA Awards $150,000 Grant to Kathryn Swoboda, MD, Massachusetts General Hospital
- Biogen Releases Community Statement on Spinraza Access and New Data
- Cure SMA Awards $150,000 Grant to Chad Heatwole, MD, University of Rochester
- Invitae Partners with Biogen to Offer Free Genetic Testing for SMA
- Dr. Richard Finkel, M.D., Honored with the American Academy of Neurology’s 2018 Sidney Carter Award in Child Neurology
- The Discovery of Spinraza
- There is Still Time to Complete Cure SMA’s 2018 Community Survey
- Biogen Presents New Data at the American Academy of Neurology (AAN) Annual Meeting
- Spring 2018 Issue of Compass Now Available Online
- Genentech to Present New RG7916 Data at the American Academy of Neurology (AAN) Annual Meeting
- Cure SMA Awards $150,000 Grant to Bakri Elsheikh, MD, Ohio State University Wexner Medical Center
- Cure SMA Database Findings Published in the Journal of Neuromuscular Diseases
- Novartis Agrees to Acquire AveXis
- Cure SMA Awards $150,000 Grant to Brunhilde Wirth, PhD, Institute of Cologne, Germany
- Cure SMA Awards $50,000 Grant to Rashmi Kothary, PhD, University of Ottawa
- Scholar Rock Granted Orphan Drug Designation by the FDA for SRK-015 for the Treatment of Patients with SMA
- Cure SMA Awards $150,000 Grant to Robin Parks, PhD, University of Ottawa
- Cure SMA Awards $150,000 Grant to Charlotte Sumner, MD, Johns Hopkins University
- Record Number of Abstracts Submitted for 2018 SMA Researcher Meeting
- Roche Releases Community Statement on RG7916 and Olesoxime Programs
- Cure SMA Awards $75,000 Grant to Robert Kalb, MD, Northwestern University
- Cure SMA Awards $150,000 Grant to Umrao Monani, PhD, Columbia University
- Biogen Announces New Interim Phase 2 Results From NURTURE
- Cure SMA Launches Second Annual Community Survey to Address Important Issues in SMA Treatment
- Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, Medical College of Wisconsin
- Phase 2 Clinical Trial Results for CK-2127107 Expected in Second Quarter of 2018
- Biogen Releases Statement on CHERISH Data and Plans for New Clinical Research
- Winter 2018 Compass Now Available Online
- Preliminary Data from FIREFISH Trial in Type 1 SMA Infants Presented at the International Scientific Congress on Spinal Muscular Atrophy
- AveXis Releases Community Statement on Expanded Clinical Trials
- SMA Industry Collaboration Releases Spinal Muscular Atrophy Voice of the Patient (VoP) Report
- 2018 Updated SMA Drug Pipeline Released
- AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy
- Scholar Rock Announces Plan to Bring Possible SMA Treatment into Clinical Trials
- Cure SMA Announces Funding for Care Centers Across the US
- Biogen and Ionis Enter Into New Collaboration to Identify Novel Therapies for the Treatment of Spinal Muscular Atrophy
- 2017 Research Year-in-Review
- AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101
- Cure SMA-Funded Research Results in 21 Published Journal Articles
- Cytokinetics Publishes Clinical Trial Data for CK-2127107
- Cure SMA-Funded Researcher, Stephen Kolb, Publishes Paper
- Biogen Issues Community Update on Spinraza
- 2017 World Muscle Society Congress Recap
- Fall 2017 Issue of Compass Now Available
- Cure SMA Announces Over $5 Million in New Research and Care Funding
- Genentech/Roche Releases Clinical Trial Update for SUNFISH (RG7916)
- Cure SMA Contributes Article to a Special Issue of the Journal Gene Therapy
- Novartis Releases Update on LMI070 (Branaplam) Clinical Trial
- 2017 SMA Researcher Meeting Summary: Clinical Research Studies for SMA
- 2017 Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Disease Modifiers
- Victor Dubowitz Honored at 2017 Researcher Meeting for Early Contributions to the SMA Field
- Cure SMA-Funded Researchers, Diane Murrell and Deborah Boroughs, Publish Papers
- 2017 Researcher Meeting Summary: Regulation of Splicing and SMN Protein Expression
- 2017 SMA Researcher Meeting Summary: SMA Therapy Development
- 2017 SMA Researcher Meeting Summary: Special Session on Fatigue and Muscle Weakness in SMA
- Summer 2017 Issue of Compass Now Available
- Summer 2017 Update to SMA Drug Pipeline is Released
- Cure SMA Funded Standard of Care Paper Published in Neuromuscular Disorders Journal
- AveXis Announces Alignment with the FDA on Company’s GMP Commercial Manufacturing Process for AVXS-101
- Cure SMA Awards $140,000 Grant to Oliver Gruss, PhD, Rheinische Friedrich-Wilhelms-Universitat Bonn and Utz Fischer, PhD, Julius-Maximilians-Universitat Wurzburg, Germany
- Cure SMA Awards $140,000 Grant to Alberto Kornblihtt, PhD, Universidad de Buenos Aires, Argentina
- Cure SMA Awards $75,000 Grant to Yong-Chao Ma, PhD, Ann & Robert H. Lurie Children’s Hospital of Chicago
- Cure SMA Awards $75,000 Grant to Stephen J. Kolb, PhD, Ohio State University
- Cure SMA Awards $75,000 Grant to Chris Lorson, PhD, University of Missouri
- Cure SMA Announces Increase in Basic Research Funding, Now Exceeding $1 Million in Grants
- Cure SMA Awards $140,000 Grant to Christine DiDonato, PhD, Ann & Robert H. Lurie Children’s Hospital of Chicago
- Cure SMA Awards $75,000 Grant to Jean Giacomotto, PhD, University of Queensland, Australia
- Cure SMA Awards $30,000 Grant to Remy Bordonne, PhD, CNRS-Centre National de Recherche Scientifique, France
- Record Number of Abstracts Submitted for the 2017 SMA Researcher Meeting
- Cure SMA Awards $140,000 Grant to Joceyln C̫t̩, PhD, University of Ottawa
- Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University
- Biogen Presents New ENDEAR Data Showing SPINRAZA (nusinersen) Significantly Reduces Risk of Death or Permanent Ventilation
- Cure SMA Funding Results in 16 New Research Publications
- 2016 Research Year-in-Review: An Historic Year for Our Community
- The Lancet Publishes Data from Phase 2, Open-Label Trial of Spinraza (Nusinersen) in Infants
- Cure SMA-Funded Researcher, Chad Heatwole, Publishes Paper on Clinical Trial Outcomes in Adults with SMA
- Over 35 Cure SMA-Funded Researchers Contribute to Comprehensive SMA Encyclopedia
- Registration is Now Open for the 2017 SMA Researcher Meeting
- Spotlight Innovation Launches Development of STL-182 to Treat SMA
- Cure SMA-funded Researcher Sara Custer Publishes Paper
- Roche Launches New Phase 2 Clinical Trials
- Catch Up on Research News from the 2016 Family Friendly Research Poster Session
- Roche Provides New Update on Clinical Development of RG7916
- 2016 SMA Researcher Meeting Summary: Regulation of SMN Protein Expression and Function
- 2016 SMA Researcher Meeting Summary: SMA Therapy Development
- Summer 2016 Issue of Compass Now Available
- 2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 2
- 2016 SMA Researcher Meeting Summary: Identification of Candidate Therapeutic Targets and Modifiers, Part 1
- Cure SMA Recognizes Judith Melki on the Anniversary of the Discovery of the SMN Gene
- 2016 SMA Researcher Meeting Summary: Clinical Research Studies
- Cure SMA-Funded Researcher Chris Lorson Publishes Paper in Molecular Therapy
- AveXis Reports Second Quarter 2016 Interim Data from Ongoing Phase 1 Trial of AVXS-101 (Gene Therapy)
- 2016 SMA Researcher Meeting Summary: The Changing Landscape of SMA
- Clinical Trial of CK-2127107 in Patients with Spinal Muscular Atrophy
- Important Milestone Reached with First New Drug Program for SMA Advancing Towards Approval with the FDA
- Cure SMA Announces $2.5 Million in New Planned Research Funding
- Cure SMA-Funded Researcher Rashmi Kothary Publishes Paper in Scientific Reports
- Adelphi Values’ RO7350A Interview Study
- Cure SMA Awards $50,000 Drug Discovery Grant to Barrington Burnett, PhD, at Uniformed Services University of the Health Sciences
- Cure SMA Funds $2 Million in New Research in 2015-2016
- Cure SMA Awards $304,000 Drug Discovery Grant to Livio Pellizzoni, PhD, at Columbia University
- Cure SMA Receives 2016 NIH Grant
- Cure SMA Releases June 2016 Update to the SMA Drug Pipeline
- Spring 2016 Issue of Compass Now Available
- Cure SMA Awards $300,000 Drug Discovery Grant to Charlotte Sumner, in Collaboration with Imago Pharmaceuticals
- Cure SMA Releases Full Schedule for the 2016 Annual SMA Conference
- Cure SMA Awards $70,000 Grant to Christine DiDonato, Lurie Children’s Hospital of Chicago
- AveXis Reports Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1
- Cure SMA Announces 2016 Family Friendly Poster Session Presenters
- Ionis Releases Data Update from Phase 2 Study in Infants with SMA
- Annual SMA Conference to Host Panels on NDA Process and SMA Drugs in Development
- Cure SMA-Funded Researchers Receive NIH Grant
- Cure SMA Awards $30,000 Grant to Jocelyn Cote, PhD, University of Ottawa
- Cure SMA Awards $140,000 Grant to Arthur Burghes, PhD, The Ohio State University
- Cure SMA Awards $90,000 Grant to Antoine Cl̩ry, PhD, ETH Zurich
- Cure SMA Awards $140,000 Grant to Christine Beattie, PhD, Ohio State University
- Record Number of Abstracts Submitted for the 2016 SMA Researcher Meeting
- Cure SMA-Funded Researchers Publish Paper on Motor Neuron and Astrocyte Defects
- Patients and Caregivers of Patients with Type II and III SMA Needed for Educational Research Survey
- Cure SMA Awards $140,000 Grant to Rashmi Kothary, MD, Ottawa Hospital Research Institute
- Cure SMA Awards $140,000 Grant to Megerditch Kiledjian, PhD, of Rutgers
- Cure SMA-Funded Researcher Publishes Paper on Biomarker Study
- Abstract Submission Opens for 2016 SMA Researcher Meeting
- Cure SMA Funding Leads to 18 New Research Publications in 2015
- Cure SMA Funds 28 Research Projects in 2015, as Drug Pipeline Grows to 18 Programs
- Cure SMA Scientific Advisory Board Meets in Chicago
- Fall 2015 Issue of Compass Highlights SMA Researcher Meeting
- Cure SMA Medical Advisory Council Meets in Chicago
- Cure SMA and Other Venture Philanthropy Leaders Share Insight at Partnering for Cures Panel
- Cure SMA Publishes Journal Article on the Patient Voice
- Cure SMA-Funded Researchers Publish Paper in Journal Pediatrics
- Registration Open for the 2016 SMA Researcher Meeting
- 2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 2
- 2015 SMA Researcher Meeting Summary: SMN Partners and Targets, Part 1
- 2015 SMA Researcher Meeting Summary: SMA Pathology
- 2015 SMA Researcher Meeting Summary: Drug Discovery
- 2015 SMA Researcher Meeting Summary: Clinical Research
- 2015 SMA Researcher Meeting Summary: Newborn Screening
- 2015 SMA Researcher Meeting Summary: Emerging Trends in Motor Neuron Pathobiology
- Cure SMA Announces Nearly $2 Million in New Planned Research Funding
- Read the Summer 2015 Issue of Compass
- SMA Drug Pipeline Grows to 18 Programs
- Cure SMA Receives NIH Grant
- Collaborative Ways Cure SMA is Driving Research Results
- Cure SMA Funds $1.85 Million in New Research Grants in 2014-2015
- Cure SMA Announces Additional $445,000 for Nationwide Children’s Hospital Gene Therapy Program led by Brian Kaspar, PhD
- Spring 2015 Issue of Compass Now Available
- Annual SMA Conference to Feature Panel of Leading SMA Drug Experts
- Share Your SMA Story for a New Research Project
- Human Tissue Donation Program Advances Understanding of SMA
- Cure SMA Announces New $140,000 Basic Research Grant to Chad Heatwole, MD, at the University of Rochester
- SMA Researcher Meeting to Host Over 100 Presentations
- Biogen Announces NURTURE, a New Phase 2 Clinical Trial
- Cure SMA Announces Additional $315,000 for SMA Drug Program at The California Institute of Biomedical Research
- Cure SMA Releases Updated Overview of Research Model
- Cure SMA Hosts SMA Researcher Meeting this June
- Cure SMA Awards $140,000 Grant to Francesco Lotti, PhD, of Columbia University
- Cure SMA-Funded Researcher Publishes Paper on Critical Issues for SMA Families and Doctors
- Roche Buys Trophos to Continue Work on SMA Drug
- Cure SMA Announces New $30,000 Clinical Care Research Grant to Deborah Boroughs at BAYADA Home Health Care
- Cure SMA Awards $140,000 Grant to Sara Custer, PhD, of Indiana University
- Cure SMA Announces New $50,000 Care Research Grant to Rebecca Davis in the Lab of Dr. Swoboda at University of Utah
- Cytokinetics and Astellas Announce New Phase 2 Clinical Trial
- Cure SMA Announces New $50,000 Clinical Care Research Grant to Nilesh Mehta at Boston Children’s Hospital
- Results Published on First Large Animal Model of SMA
- Cure SMA Funding Leads to 16 New Research Publications in 2014
- Cure SMA Funds 26 Research Projects in 2014
- Isis Expands Phase 3 Clinical Trials
- SMA Drug Pipeline Continues to Grow
- Cure SMA Researchers Present at Society for Neuroscience Meeting
- PTC Therapeutics Announces New Phase 1a/2b Clinical Trial
- Isis Announces Second Phase III Clinical Trial
- Cure SMA Scientific Advisory Board Meets in Washington D.C.
- Promising Results Published on Cure SMA-Funded Gene Therapy Project
- Read the Fall 2014 Issue of Compass
- 2014 SMA Researcher Meeting Summary: Clinical Trials
- 2014 SMA Researcher Meeting Summary: Therapy Development
- 2014 SMA Researcher Meeting Summary: SMA Pathology
- 2014 SMA Researcher Meeting Summary: The SMN Protein
- Isis Releases Phase II Clinical Trial Results
- AveXis Announces the Completion of Dosing of the Low-Dose Cohort in US Clinical Trial for Spinal Muscular Atrophy
- Isis Presents Phase II Trial Results
- AveXis Receives Orphan Drug Designation
- Isis Begins Recruiting for Phase III Trial
- Cure SMA-Funded CALIBR Research Collaboration Expanded to USC
- Catch Up on Research News from our Family Friendly Poster Session
- Spinal Muscular Atrophy Biomarker Study Reaches Enrollment Goals
- RFP Closes for Spinal Muscular Atrophy Basic Research Funding
- Cure SMA Announces Basic Research Funding for Dr. Lyndsay Murray
- Cure SMA and PPMD Invite Spinal Muscular Atrophy Parents to Take Part in Two Clinical Trial Surveys
- Funding Process Reaches Next Steps for Spinal Muscular Atrophy Clinical Care Research
- Cure SMA Featured in Assay and Drug Development Technologies
- Spinal Muscular Atrophy Mice Model Results Published in the Journal Science
- Isis Begins Phase III Clinical Trial
- Cure SMA Announces New Research Funding
Support & Care
- Spinal Muscular Atrophy (SMA) Update in Best Practices: Recommendations for Treatment Considerations
- Cure SMA Hosting Puerto Rico Summit of Strength & Walk-N-Roll
- Cure SMA is Thrilled to Launch New Travel Resources Webpage and a New Travel Support Package
- Spinal Muscular Atrophy (SMA) Update in Best Practices: Recommendations for Diagnosis Considerations
- Living Unlimited with Spinal Muscular Atrophy: A Booklet for Adults with SMA
- Cure SMA Care Center Network Site Expansion
- Cure SMA Support Services Now Expanded to Individuals and Families in Puerto Rico
- International Day of Women and Girls in Science
- Understanding the Benefits of Aqua Therapy
- Cure SMA Launches New Teen & Adult Independence Assistance Package – Check It Out Today!
- Cure SMA Presents Data on Cure SMA Care Center Network and SMA Clinical Data Registry
- Cure SMA Launches LifeVac Support Program to Help in Choking Event
- Cure SMA Launches Virtual Therapy Program for Adults with SMA
- Cure SMA Career Panel Webinar Series to Launch in February
- Cure SMA Announces 2021 Summit of Strength Program Events
- Cure SMA Launches Physical Therapy Webinar Series
- Cure SMA to Host Webinar Series on Wellness for the SMA Community
- Now Available! New COVID-19 PPE Package from Cure SMA
- Cure SMA Announces Two New Virtual Programs for Adults with SMA
- Summit of Strength Virtual Webinar Series Continues Its Success!
- Cure SMA Immunizations Recommendations During Flu Season and Amidst the COVID-19 Pandemic
- NeurologyLive Article Now Available, Featuring an Interview with Cure SMA’s Dr. Mary Schroth
- Cure SMA Publishes Paper in Partnership with NeurologyLive on Impact of COVID-19
- Cure SMA Launches New Medical Alert Bracelet Support Program
- Cure SMA to Meet with FDA for Critical Path Innovation Meeting
- Cure SMA Announces Summit of Strength Virtual Webinar Series
- Cure SMA Launches New COVID-19 Support Package
- COVID-19 Assistance Program Available to SMA Community
- Cure SMA Adds New Equipment Item for Teens and Adults with SMA
- Summit of Strength Program Dates Confirmed
- Cure SMA Awards Grants to Four States to Expediate the Implementation of SMA Newborn Screening
- Update on Cure SMA Summit of Strength Program – Gearing Up for 2020!
- Check Out the Cure SMA Booklet on “Scientific Considerations for Drug Combinations”
- 2019 Summit of Strength Program Continues its Success!
- Cure SMA Newborn Screening Registry (NBSR)
- Zolgensma Update and Approval Webinars Now Available Online
- Cure SMA Awards Grants to Four States to Expediate the Implementation of SMA Newborn Screening
- Cure SMA Receives $150,000 Grant From the Oscar G and Elsa S Mayer Family Foundation
- Cure SMA Publishes Journal Article on Benefit-Risk Preferences in SMA
- Cure SMA and Genentech Partner to Provide Travel Support to Adults with SMA the Annual SMA Conference
- Cure SMA Grants More Than 900 Scholarships for Annual SMA Conference
- Cure SMA Care Center Network Expands to 10 Sites
- 2019 Annual SMA Conference Registration Continues to Grow!
- What is the Summit of Strength Program?
- We’re Celebrating Local Support
- Cure SMA and Parent Project Muscular Dystrophy Announce Collaboration
- New Dates and Locations Announced for 2019 Summits of Strength
- Show Your Rare. Show You Care.
- Fall 2018 Issue of Directions Now Available
- Teen and Young Adult Survey Extended Through January 21st
- Fall 2018 Issue of Compass Now Available
- Register for Cure SMA’s Webinar on SMA Treatments and Clinical Trials
- Cure SMA is Thrilled to Launch a New Support Program for Teens and Adults with SMA
- Summit of Strength Program Receives Positive Feedback and Increases Attendance
- Cure SMA Receives Generous Funding from Luke 18:1 Foundation
- SMA Care Center Network and Clinical Data Registry Launched
- Cure SMA Launches a Family Support App for Your Mobile Device!
- Cure SMA’s Approach to Newborn Screening
- Sweet Baby Zane Funds New Equipment Pool Item
- Cure SMA Receives Generous Donation from the Cash Scanlon-Philips Foundation
- Updated 2018 SMA Standards of Care Statements Available Online
- Summit of Strength Program Kicks Off in Cincinnati
- Dr. John Kissel, Chair of Neurology at OSU Wexner Medical Center, Retires After 40 Years
- Spring 2018 Issue of Directions Now Available
- Cure SMA and Cytokinetics Announce National Platinum Partnership for 2018
- Uniting the SMA Community Through Local Programs and Support
- Invitae Partners with Biogen to Offer Free Genetic Testing for SMA
- Registration for the 2018 Annual SMA Conference Closes in Two Weeks
- Cure SMA Welcomes Back Biogen as a National Premier Partner for 2018
- House and Senate Vote to Increase Federal Funding for Newborn Screening Programs
- Cure SMA to Host Inaugural Clinical Care Meeting
- Cure SMA Welcomes New Medical Advisory Council Committee Members
- Last Week’s Webinar on Treatment Access and Clinical Trials Now Available
- Cure SMA Receives Generous Gift
- Additional Funding for SMA Care Centers Announced
- Cure SMA Announces Local Educational Symposiums in 16 US Locations
- Winter 2018 Compass Now Available Online
- Cure SMA Announces Funding for Care Centers Across the US
- Cure SMA Assists Hundreds of Families in 2017
- Miller McNeil Woodruff Foundation Awards Cure SMA Generous Gift
- Fall 2017 Directions Now Available for Download
- Adult Scholarships Offered for the 2018 Annual SMA Conference
- Cure SMA Announces Over $5 Million in New Research and Care Funding
- Recording of the September Webinar on Spinraza Updates Now Available
- Applications for Cure SMA Medical Advisory Council Due October 15th
- Cure SMA Receives Donation from the Bugenske Family
- Cure SMA Funded Standard of Care Paper Published in Neuromuscular Disorders Journal
- Cure SMA Receives Generous Donations from Fighting for Kaiden Foundation
- American College of Obstetricians and Gynecologists Recommends Expanded SMA Carrier Screening
- Cure SMA Releases Care Series Booklet on Insurance
- Cure SMA Assists Hundreds of Families in 2016
- Biogen Resources for the SMA Community
- Jadon’s Hope Foundation Donates 15 Items to Cure SMA Equipment Pool
- Adult Scholarships Offered for the 2017 Annual SMA Conference
- We’re Partnering With The Mighty!
- Adelphi Values’ RO7350A Interview Study
- Winter 2015 Issue of Directions Now Online
- Cure SMA Receives Generous Donation from Fighting for Kaiden Foundation
- Cure SMA Reaches Thousands of Families in 2015
- Cure SMA Medical Advisory Council Meets in Chicago
- Jadon’s Hope Foundation Donates 10 New Tomato Feeder Seats
- Dr. Mary Schroth Joins Cure SMA as Medical Professional Education Consultant
- Spring 2015 Directions Posted Online
- Cure SMA Publishes New Care Series Booklet on Musculoskeletal System
- Cure SMA-Funded Researcher Publishes Paper on Critical Issues for SMA Families and Doctors
- Join Cure SMA in Celebrating the International Day of Acceptance
- Bugenske Family Donates $17,000 to Cure SMA Equipment Pool
- Fall 2014 Issue of Directions Now Online
- Cure SMA Launches Survey on Family Support Programs
- Registration Opens for the 2015 Annual SMA Conference
- Applications Open for 2015 Annual SMA Conference Scholarships
- Cure SMA Provides Record Number of Resources to Families Worldwide
- Cure SMA Welcomes New Medical Advisory Council Committee Members
- Kinetic Sand Donated for Care Packages
- Medical Advisory Council Gathers for Fall 2014 Meetings
- Join Us in Kansas City
- Cure SMA Announces New Subcommittee on Adults with SMA
- Applications for the Cure SMA Medical Advisory Board Due 10/1/14
- Funding Process Reaches Next Steps for Spinal Muscular Atrophy Clinical Care Research
- Cure SMA Reaches Out to Newly Diagnosed Families
Uncategorized
- Make an Impact with Cure SMA’s Mission, Milestones, Momentum Campaign
- Fall 2024 Walk-n-Roll Registration is Open
- Registration is Open for the 2024 Annual SMA Conference!
- Biohaven Completes Enrollment in Pivotal Phase 3 Study
- The State of SMA – Toward Improved Therapeutics and Care
- Thank You for an Amazing 2023 Annual SMA Conference!
- Cure SMA Applauds Federal Agency Decision that Should Lead to Better Insurance Coverage for Seat Elevation Systems in Power Wheelchairs
- Biogen Announces New Updates Across its SMA Research Program at 2023 MDA Conference
- Cure SMA Announces Expanded Phase 8 of SMA Industry Collaboration
- Cure SMA Welcomes New Care Center Network Site!
- FDA Issues Safety Warning Regarding Neck Float Devices for Babies
- Cure SMA Publishes Results from Survey Measuring Disease Burden in Adolescents and Young Adults
- Cure SMA Publishes Research Exploring What Is Important to SMA Clinical Trial Participants
- Financial Planning with a Disability
- Community Spotlight: Nikki McIntosh
- Community Spotlight: Scurria Family
- 2019 SMA Researcher Meeting Summary: Clinical Drug Development
- What to Expect in a Clinical Trial
- Cure SMA Statement on Tax Reform Bill and the Repeal of the Individual Mandate
- Cure SMA Announces $5.05 Million in Research Funding for FY18
- Biogen Completes Rolling Submission of New Drug Application to FDA