An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

The FDA said it was clearing the way for Sarepta Therapeutics to resume shipments of its gene therapy for Duchenne muscular ...

Genethon is determined to bring GNT0004 to market for young patients and their families who are waiting for a therapeutic ...

Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

On Thursday 28 August, Sam Taylor, 46, his wife Sue, 47, and daughter Beth, 17, from Crooklands in Cumbria will set off to ...

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...

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