Latest News
Novartis Gene Therapies SMA Community Update
Dear SMA Community, It was a pleasure to be able to join so many of you at the Cure SMA Annual Conference in Orlando this year, and we look forward […]
Read More ›Cure SMA on Capitol Hill – A Message from Allie Williams
Hey, it’s Allie here from Oklahoma! I am privileged to serve on the Cure SMA Adult Advisory Council and am part of a group of adults and family members with […]
Read More ›SMA Highlights and Updates from Biogen
Dear SMA Community, As we reflect on SMA Awareness Month, we take this opportunity to celebrate all those who are a part of the SMA community and the future we’re […]
Read More ›Identifying Biomarkers of Spinal Muscular Atrophy for Further Development
An important part of Cure SMA’s core mission is to carry out and support research that will improve the quality of life for all people with SMA. We recently published […]
Read More ›NMD Pharma Initiates Phase II Trial for SMA
NMD Pharma, a clinical-stage biotech company leading the development of novel first-in-class therapies for severe neuromuscular disorders, recently announced that the first SMA patient has been dosed in their Phase […]
Read More ›Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial
Today, Scholar Rock announced that enrollment in their Phase 3 SAPPHIRE trial is now complete. The Phase 3 trial will evaluate the safety and efficacy of apitegromab in nonambulatory patients with […]
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