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

A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular ...

Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the ...

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

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

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

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

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

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

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

MyHealthTeam, a Swoop company, the creator of one of the largest, engaged patient social networks in healthcare, has announced the launch of myMDteam. This new website and patient community is ...

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