November 2025

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 (onasemnogene abeparvovec-brve) is a gene therapy that works by delivering a functional copy of the SMN1 gene directly to motor neurons, helping the body restore […]

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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) for apitegromab, a potential treatment for children and adults with SMA.   Cure SMA was present and active at this FDA meeting to represent our

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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 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

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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 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

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