Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA) New Phase 1b data further illustrate potential of salanersen in SMA, ...

The company discontinued developing emugrobart after two studies showed the drug didn’t achieve intended outcomes.

The main beneficiary of Roche’s discontinuation of an investigational spinal muscular atrophy drug is Scholar Rock, which was ...

Please provide your email address to receive an email when new articles are posted on . Interim results also showed reduction of the neurodegenerative biomarker neurofilament light chain. Serious ...

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies ...

The big pharma company will no longer progress emugrobart to late-stage trials in FSHD and SMA due to a lack of efficacy.

Please provide your email address to receive an email when new articles are posted on . The study included 58 individuals with SMA types 2 and 3 who were treated with IV apitegromab for 52 weeks. At ...

Scholar Rock Holding Corp. (NASDAQ:SRRK) said on Sunday the company will present new efficacy and safety data from the Phase 3 pivotal SAPPHIRE trial at the 2025 Muscular Dystrophy Association (MDA) ...