The Health Resources and Services Administration is requesting public comments on its recommendation to add metachromatic leukodystrophy and Duchenne muscular dystrophy to the Recommended Uniform ...

DYNE-251 received FDA breakthrough therapy designation, showcasing its potential for patients with Duchenne muscular ...

A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular ...

Jefferies analysts said these detailed safety outcomes confirm the gene therapy's positive risk/benefit profile in ambulatory ...

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

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

After two patients who received the investigational CDC7 blocker died, pushing forward with SGR-2921’s development would be ...

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 well-connected drug company and Laura Loomer wanted Kennedy ally Vinay Prasad gone. Trump Chief of Staff Susie Wiles got ...

Trump loyalist Laura Loomer is openly campaigning to blunt Health and Human Services Secretary Robert F. Kennedy Jr.’s ...

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

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