Latest News
Novartis Receives FDA Approval of Itvisma for the Treatment of SMA
Novartis today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Itvisma (onasemnogene abeparvovec-brve) to treat adults and children 2 years of age and older with spinal muscular atrophy (SMA). Itvisma […]
Read More ›Scholar Rock and Cure SMA Share Update on Apitegromab and FDA Meeting
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) […]
Read More ›Novartis Releases Fall SMA Community Update Letter
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 […]
Read More ›Unlock a Future of Possibilities: Cure SMA Launches Year-End Campaign
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 […]
Read More ›Scholar Rock Releases SMA Community Update Letter on Apitegromab
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 […]
Read More ›Biogen to Initiate Phase 3 Pivotal Study Evaluating Investigational Drug Salanersen
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 […]
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