US biotech major Biogen has presented additional results from the Phase Ib study of salanersen, an investigational novel ...
New Phase 1b data support the safety and effectiveness of salanersen over one year in children with SMA who had the potential for improvement due to suboptimal clinical status with prior gene ...
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 ...
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, ...
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) ...
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said in a statement ...
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic ...
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