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

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

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.

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

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

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

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

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

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

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