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 ...

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

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 ...

The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla.

The U.S. Food and Drug Administration (FDA) announced on Friday, after market close, that it is investigating the death of an ...

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