Read the Scholar Rock community statement here. Scholar Rock recently held an in-person Type A meeting with the FDA on November 12 to discuss the Biologics License Application (BLA) for apitegromab, a potential treatment for children and adults with SMA. Cure SMA was present and active at this FDA meeting to represent our
Scholar Rock and Cure SMA Share Update on Apitegromab and FDA Meeting Read More »
Novartis recently released an update on their continued collaboration with the SMA community through fundraising events and community support programs, and they will continue to provide timely updates as they become available. Read the full community update letter here. “Over the past few months, we’ve been inspired by the continued strength, unity, and advocacy across
Novartis Releases Fall SMA Community Update Letter Read More »
As 2025 starts to wind down, Cure SMA is proud to launch our year-end fundraising campaign: Unlock a Future of Possibilities. This theme represents hope, progress, and the life-changing difference your support makes for individuals and families impacted by spinal muscular atrophy (SMA). At the heart of this campaign are four members of the SMA community
Unlock a Future of Possibilities: Cure SMA Launches Year-End Campaign Read More »
Yesterday, Scholar Rock shared an SMA community update letter on their Biologics License Application (BLA) for apitegromab in SMA. As shared on September 23, 2025, Scholar Rock received a Complete Response Letter (CRL) from the FDA for the apitegromab BLA in SMA. As a reminder, the CRL was solely related to manufacturing observations identified at
Scholar Rock Releases SMA Community Update Letter on Apitegromab Read More »
Today, Biogen announced plans to initiate Phase 3 development to evaluate the effectiveness and safety of salanersen, an investigational drug for the treatment of spinal muscular atrophy (SMA). Phase 3 initiation follows the announcement of positive interim results of the Phase 1 study of salanersen, which informed the design of the studies. Biogen is preparing
Biogen to Initiate Phase 3 Pivotal Study Evaluating Investigational Drug Salanersen Read More »
