November 2025

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

(opens in new tab) 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,