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 ...

K.S.R.A. Praneeth, an 18-year-old battling Duchenne Muscular Dystrophy, competed in the World IPCA Chess Championship in Goa, ...

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

A 12-year-old boy from Carthage, Tennessee finds joy in his power wheelchair. His family are hoping for a cure for his muscular disease, Duchenne.

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

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

Novartis has made a takeover approach for rare disease-focused biotech Avidity Biosciences, as Big Pharma continues to hunt ...

Collaboration Revenue: Collaboration revenue was $2.0 million for the second quarter of 2025, compared to $94.7 million for the same period in 2024. This decrease is primarily attributable to the ...

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 ...

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

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