TreatmentsJan 13, 2026
High-Dose Regimen of nusinersen receives European Commission Approval for the treatment of SMA
High-Dose Regimen of nusinersen receives European Commission Approval for the treatment of SMA.
SMA Newsroom
TreatmentsNov 25, 2025
Breaking news: FDA approval for Itvisma® (onasemnogene abeparvovec-brve), the intrathecal formulation of onasemnogene abeparvovec.
Today, Novartis announced that the company has received the FDA approval for Itvisma® (onasemnogene abeparvovec-brve), the intrathecal formulation of onasemnogene abeparvovec, to treat SMA in adult and pediatric patients 2 years of age and older with a confirmed mutation in SMN1.
Community Update Roche: EMA approval of the risdiplam tablet
We are pleased to share a community letter from Roche, announcing that the European Commission (EC) has approved a new, room- temperature stable tablet formulation of risdiplam.
TreatmentsMar 28, 2025
Community update from Scholar Rock
Scholar Rock announced that the FDA has granted priority review for the Biologics License Application for apitegromab, an investigational muscle-targeted treatment developed to improve motor function for people living with SMA who are receiving an SMN-targeted treatment.