Author name: Sarah McCall

Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate

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Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before 6 weeks of age, with most achieving motor milestones similar to children without SMA All children were able to swallow and feed orally, with none requiring permanent ventilation Evrysdi is the only non-invasive SMA therapy and is approved in over 100 countries, […]

Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate Read More »

Cure SMA Hosting Puerto Rico Summit of Strength & Walk-N-Roll

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Cure SMA is thrilled to announce the 2025 San Juan, Puerto Rico Summit of Strength,taking place on Saturday, February 1. The Summit of Strength is a community educational program hosted by Cure SMA. These in-person events provide attendees the opportunity to learn from an array of national and local experts surrounding topics that are valuable to

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Cure SMA is Thrilled to Launch New Travel Resources Webpage and a New Travel Support Package

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Along with funding SMA research and care, Cure SMA provides thousands of individuals and families with vital support and resources that help people navigate life with SMA.  Travel should be accessible for everyone but we know that is not often the case. With this in mind, Cure SMA is thrilled to launch some new travel

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New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA

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Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in both individuals previously treated and treatment-naïve to nusinersen Investigational regimen also shows more rapid slowing of neurodegeneration, as measured by neurofilament Biogen plans to submit regulatory applications around the world

New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA Read More »

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

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

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

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

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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