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 ...

On Saturday 12 July, Claire Ellis, 34, a teacher from Doncaster set off from her home to cycle 100km in support of her son ...

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.

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, ...

Last month Claire Ellis, aged 34, a teacher from Doncaster, set off from her home to cycle 100km in support of her son Declan ...

Vinay Prasad, a former critic of U.S. COVID-19 policies, returns to the FDA's Center for Biologics Evaluation and Research after briefly leaving. His tenure drew criticism, notably over the handling ...

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

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

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

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