Front Page News
Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate
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 […]
Read More ›Cure SMA Hosting Puerto Rico Summit of Strength & Walk-N-Roll
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 […]
Read More ›Cure SMA is Thrilled to Launch New Travel Resources Webpage and a New Travel Support Package
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 […]
Read More ›New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA
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 […]
Read More ›Scholar Rock Announces Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA)
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 […]
Read More ›Celebrating Disability Pride and the 34th Anniversary of the ADA at the White House
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 […]
Read More ›Biogen Announces Positive Topline Results from Study of Higher Dose Regimen of Nusinersen, Showing Significant Benefit in Treatment of SMA
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 […]
Read More ›Community Spotlight: Viola Dwyer, SMA Community Advocate from Pennsylvania
During this SMA Awareness Month, Cure SMA recognizes all SMA community advocates – individuals with spinal muscular atrophy (SMA) and their families – who are […]
Read More ›Cure SMA Attends Spring 2024 Professional Organization Conferences to Promote Findings from Real World Evidence Collaboration Initiatives
Cure SMA is pleased to announce the involvement of scientific leadership, in the 4th Scientific International Congress on SMA (March 14 – 16, 2024), the […]
Read More ›Cure SMA Launches Request for Proposals for SMA Research Projects and Postdoctoral Fellowships
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 […]
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