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 ...

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.

Now, maybe, it’s déjà vu all over again as the FDA is reportedly considering rejecting the company’s request to fast-track what could be a vital new gene therapy called SRP-9001, or, if you ...

The US Food and Drug Administration (FDA) has approved vamorolone oral suspension (Agamree, Santhera) for the treatment of Duchenne muscular dystrophy (DMD) in patients as young as age 2 years ...

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 ...

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.