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

The Trump FDA tries to kill a therapy that has helped boys with a deadly diagnosis.

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

The U.S. FDA is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta (SRPT) Therapeutics gene therapy for Duchenne ...

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

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

Duchenne muscular dystrophy patients Jack Willis (center), Nolan Willis (right) and Max LeClaire, attended the opening of Sarepta Therapeutics new headquarters in Cambridge, Mass., in 2014.

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 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.