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.
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.
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.
Roche shared updates on the final results of the now-concluded SUNFISH study, which assessed the efficacy and safety of risdiplam in people living with SMA aged 2-25 years.