Latest News
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. […]
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 of this investigational dose regimen. […]
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 raising awareness about SMA and […]
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 2024 International Society for Pharmacoeconomics […]
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