Scholar Rock submitted its BLA of apitegromab for the treatment of patients with Spinal Muscular Atrophy with a request for Priority Review to be given. Explore more details here.

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

Scholar Rock (SRRK), a late-stage biopharmaceutical company developing innovative treatments for spinal muscular atrophy, ...

Learn more about whether Ionis Pharmaceuticals, Inc. or PTC Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

Problems with chemical messengers like dopamine were tied to posture issues in SMA in a mouse study, but levodopa therapy may ease symptoms.