Latest 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 without SMA All children were […]
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 community educational program hosted by […]
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 with SMA. Travel should be […]
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 both individuals previously treated and […]
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 Rock, a late-stage biopharmaceutical company […]
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 reception at the White House. […]
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