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

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

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

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

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

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

Liquidating its stake in Arrowhead helps Sarepta cover a milestone payment and secure what one analyst described as ...

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

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.

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 ...

A handful of drug companies have formed a group to present lawmakers with research on the negative impacts of Medicare drug ...