Sarepta Therapeutics SRPT announced top-line data from part 2 of the mid-stage study — SRP-9001-102 (Study 102) — evaluating its lead gene therapy candidate, SRP-9001, in children with Duchenne ...

Duchenne muscular dystrophy ("DMD") is a rare genetic disease and a severe form of muscular dystrophy, primarily affecting males. It progressively weakens and degenerates skeletal and heart muscles.

Research and Markets has announced the addition of the "Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review, 2017" report to their offering. This report provides an overview ...