Early trial data suggest RNA therapies SRP-1001 and SRP-1003 reach muscle and show biomarker activity in muscular dystrophy.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a ...
Myotonic dystrophy is a condition that causes thinned muscles, decreased muscle tone, and muscle weakness. Over time, a person may lose their strength and have issues relaxing their muscles. The ...
A person may inherit the genetic changes responsible for muscular dystrophy. These genetic changes can also occur due to spontaneous genetic mutations. In either case, the disease is not preventable.