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.
We are pleased to share the latest Community Letter issued by Roche, providing an update on their ongoing efforts in Spinal Muscular Atrophy (SMA) in 2024. The letter includes the latest developments on risdiplam and Roche’s SMA clinical development program.