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 ...

Data from the DELIVER registrational expansion cohort is expected in late 2025, with a potential BLA submission for U.S. accelerated approval anticipated in early 2026 - WALTHAM, Mass., Aug. 04, 2025 ...

Dyne Therapeutics' DYNE-251 receives FDA breakthrough status for Duchenne muscular dystrophy treatment. Read more here.

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

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

Precision BioSciences Inc., a clinical-stage company developing gene editing therapies for diseases with high unmet need, has ...

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

Sarepta’s Elevidys is back on the market for ambulatory patients with Duchenne muscular dystrophy, Health Secretary Robert F.

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

Wave Life Sciences (WVE) stock gains as Canaccord Genuity launches its coverage with a Buy, citing multiple catalysts for the ...