Author name: Jesse Aynes

Yesterday, Scholar Rock shared an SMA community update letter on their Biologics License Application (BLA) for apitegromab in SMA. As shared on September 23, 2025, Scholar Rock received a Complete Response Letter (CRL) from the FDA for the apitegromab BLA in SMA. As a reminder, the CRL was solely related to manufacturing observations identified at […]

Today, Biogen announced plans to initiate Phase 3 development to evaluate the effectiveness and safety of salanersen, an investigational drug for the treatment of spinal muscular atrophy (SMA). Phase 3 initiation follows the announcement of positive interim results of the Phase 1 study of salanersen, which informed the design of the studies. Biogen is preparing