NMD Pharma recently shared exciting news with the SMA community: the last participant has completed their part in the SYNAPSE-SMA Phase 2 clinical trial, testing ignaseclant (formerly known as NMD670), an investigational therapy designed to help strengthen skeletal muscles. The trial included 52 adults with SMA Type 3 at sites across the US and Europe. […]

Today, Scholar Rock shared that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational treatment for spinal muscular atrophy (SMA), with a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2026. This is a meaningful step forward in the path toward a potential new

Cure SMA is pleased to announce the launch of Phase 11 initiates for the SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration, we