As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s request, which was issued Friday.

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

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

After decades of research, gene therapy has been approved for Duchenne Muscular Dystrophy, a degenerative muscle disease diagnosed each year in about 1 in 3,500 newborns, almost all boys.

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

Global Duchenne Muscular Dystrophy Drugs Market Report 2023: Sector is Expected to Reach $4 Billion by 2028 at a CAGR of 11.88% - ResearchAndMarkets.com May 18, 2023 07:21 AM Eastern Daylight Time ...

An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...

Duchenne Muscular Dystrophy may no longer be a death sentence. Oct. 17, 2012— -- Although he made it to 29 years old with Duchenne muscular dystrophy, Jason Williams was not expected to ...

The Food and Drug Administration Monday approved the first drug to treat Duchenne muscular dystrophy, a rare and lethal muscle weakening disorder that affects about 15,000 Americans.

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.