SMA Newsroom

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.

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. 

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.

We are proud to announce the five scientific projects that have been selected for funding through our Call for Research no. 12.