SMA Newsroom

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.

Novartis shared updates on the results from Phase III STEER and Phase IIIb STRENGTH studies, evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in people living with SMA aged two to <18 years.