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 ...

Taking a break from running the halls of Sammamish High School in a game of catch-me-if-you-can, five-year-old Aiden Leffler climbs on the lap of his new f ...

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

Genetic Disorder Cluster Muscular Dystrophy Drug Development Pipeline Review 2017 - 108 Products in Development for DMD, by 57 Companies and Nine Academic Institutions - Research and Markets ...