Author name: Sarah McCall

Scholar Rock Announces Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA)

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Today, Scholar Rock announced plans to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorisation application (MAA) in Q1 2025. Scholar Rock, a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today […]

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Celebrating Disability Pride and the 34th Anniversary of the ADA at the White House

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This week, Cure SMA and advocates with SMA celebrated Disability Pride and the 34th Anniversary of the Americans with Disabilities Act (ADA) with a special reception at the White House. The event marked a significant occasion to reflect on the progress made and the challenges that remain in achieving full equality and inclusion for people

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Biogen Announces Positive Topline Results from Study of Higher Dose Regimen of Nusinersen, Showing Significant Benefit in Treatment of SMA

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The positive study demonstrates the potential for investigational higher dose nusinersen regimen to advance the treatment of SMA.  Biogen plans to submit for regulatory approval of this investigational dose regimen. Biogen today announced positive, topline data from the pivotal cohort (Part B) of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of a

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Community Spotlight: Viola Dwyer, SMA Community Advocate from Pennsylvania

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During this SMA Awareness Month, Cure SMA recognizes all SMA community advocates – individuals with spinal muscular atrophy (SMA) and their families – who are raising awareness about SMA and advocating for SMA community priorities throughout the year. Cure SMA advocates play a crucial role in shaping policy and raising awareness. Cure SMA spoke with

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Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Real World Evidence Collaboration Initiatives

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Cure SMA is pleased to announce the involvement of scientific leadership, in the 4th Scientific International Congress on SMA (March 14 – 16, 2024), the 2024 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conference (May 5 – 8, 2024), and the Annual SMA Research & Clinical Care Meeting (June 5 – 7, 2024). Our

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Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships

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Earlier this summer, at the Annual SMA Research and Clinical Care Meeting, Cure SMA highlighted six new basic research grants totalling $750,000. Cure SMA is now proud to announce another $750,000 will be awarded for additional research. With more than $85 million in funding to date in support of basic research grants and drug discovery

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August is Spinal Muscular Atrophy (SMA) Awareness Month! 

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Whether you have SMA, have a loved one with SMA, are a researcher, care provider, industry partner, or are new to the SMA community, SMA Awareness Month is a time to come together to celebrate, honor, reflect, remember, and learn. The theme of SMA Awareness Month 2024 is ‘Breakthroughs Begin with You.’ It’s an opportunity for you

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Cure SMA Publishes Update on the Birth Prevalence of Spinal Muscular Atrophy (SMA)

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Cure SMA is pleased to announce the publication of “Newborn Screening and Birth Prevalence for Spinal Muscular Atrophy in the US” in JAMA Pediatrics on July 15, 2024. This paper reports the largest data collection of infants with SMA identified by  newborn screening in the U.S. The objectives were: 1) to update the estimated birth

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Special Session Highlights from 2024 Annual SMA Research & Clinical Care Meeting

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Each year, Cure SMA collaborates with our Scientific Advisory Board to plan a Special Session to be held during the Annual Research & Clinical Care Meeting. This Special Session is designed to focus on a topic that is currently of great interest to both researchers and clinicians. This year’s Special Session, “The Importance of Understanding

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Congressional Action Recognizes Unmet Needs of SMA Community

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This week, a key congressional committee recognized the unmet, everyday living needs of individuals with spinal muscular atrophy (SMA) by urging the National Institutes of Health (NIH) to support new SMA research. The U.S. House Appropriations Committee approved a funding bill on July 10 that provides resources and direction to the NIH and other federal

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