New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...

Sarepta Therapeutics faces a challenging period as it navigates recent setbacks with its groundbreaking gene therapy, leading ...

The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment ...

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is one of the 10 Best Small-Cap Stocks to Buy According to Billionaires. On July 10, ...

FDA issued Complete Response Letter Capricor plans to resubmit its BLA to include data from the ongoing Phase 3 HOPE-3 trial ...

Farwell succeeds Jordan Dubow, who will continue to serve as a consultant and chair of the Clinical Advisory Board.

Alex Harold was diagnosed with Duchenne muscular dystrophy at age four and has long been raising funds for Muscular Dystrophy ...

Around 3,500 FDA employees received termination emails; FDA Commissioner Marty Makary suggests lowering industry user fees ...

TAS-205 showed no significant impact on motor function in patients with Duchenne muscular dystrophy (DMD), highlighting the ongoing search for effective treatments for the rare condition.

Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer ...