The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

Shares of Sarepta Therapeutics Inc (NASDAQ:SRPT) plunged 10% after the company disclosed that the U.S. Food and Drug ...

After initially refusing to suspend Elevidys distribution after two deaths, Sarepta has now given in to the FDA’s request, ...

Sarepta's pause on its prescription gene therapy drug for Duchenne muscular dystrophy gives the company time to work with the FDA on reviews. Infusions nationwide will be canceled. Also in the news, ...

If the study fails to shows it helps, the FDA said it could withdraw the drug. Duchenne muscular dystrophy is a rare disease, affecting about 1 of every 3,600 boys worldwide and usually causing ...

The Food and Drug Administration Monday approved the first drug to treat Duchenne muscular dystrophy, a rare and lethal muscle weakening disorder that affects about 15,000 Americans. The FDA’s ...

FDA’s panel of expert advisers recommended against the drug's approval after an emotional, packed meeting in which children with muscular dystrophy pleaded for the drug’s approval and doctors ...

The FDA is considering a drug aimed at just 13 percent of muscular dystrophy patients. It’s a highly tailored therapy that uses a completely new approach to treating the condition.

The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the risks and benefits of the drug, said Dr. Sharon Hesterlee, chief researcher at the association.

The Food and Drug Administration awarded priority review status for a drug intended to treat a childhood-onset form of muscular dystrophy, according to the Chicago Tribune. Northbrook, Ill.-based ...