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 ...

A teenager living with a severe and degenerative condition is hoping to retain his independence in the face of the cruel ...

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

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

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

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 ...

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

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

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

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...