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 ...

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to ...

The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

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.

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

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

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

Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy fell short of its main Phase 3 goal, but the firm contends the full body of evidence supports expanding the therapy’s label to ...

Deflazacort, a newly FDA-approved drug for Duchenne muscular dystrophy, has an $89,000-a-year price tag. It has been put on hold because of an uproar over drug prices.