Industry Updates
FDA Grants Breakthrough Therapy Designation to Biogen’s Salanersen for Spinal Muscular Atrophy
Biogen recently announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to salanersen, an investigational therapy being studied for the […]
NMD Pharma Releases Community Letter Regarding Phase 2 SYNAPSE-SMA Clinical Trial
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 […]
FDA Accepts Scholar Rock’s Biologics License Application (BLA) to Review Apitegromab for SMA
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 […]
Cure SMA Launches Phase 11 SMA Industry Collaboration
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 […]
Scholar Rock Resubmits Biologics License Application (BLA) for Apitegromab to the FDA
Today, Scholar Rock announced that it has resubmitted its Biologics License Application (BLA) to the Food and Drug Administration (FDA) for apitegromab, an investigational therapy […]
Biogen Receives FDA Approval of High Dose SPINRAZA for the Treatment of SMA
Biogen today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for the High Dose regimen of SPINRAZA (nusinersen) to […]
Evrysdi Risdiplam Tablet Is Now Approved For Broader Use In SMA
We’re excited to share an important update for the spinal muscular atrophy (SMA) community. The U.S. Food and Drug Administration (FDA) has approved a label […]
Novartis Releases Winter SMA Community Update Letter
Novartis recently shared a Winter 2026 update highlighting the FDA approval of ITVISMA® (onasemnogene abeparvovec-brve) for intrathecal use in people ages 2 and older with […]
Biogen Announces Results from the Phase 2/3 DEVOTE Study
Today, Biogen announced the publication of results from the pivotal Phase 2/3 DEVOTE study in Nature Medicine, highlighting the potential benefits of a high-dose regimen […]
Genentech Releases Winter SMA Community Update Letter
Genentech recently released an update on their continued collaboration with the SMA community and Evrysdi® tablet updates. They will continue to provide timely updates as […]