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

Less than weeks after stepping down, Prasad will return to lead CBER “at the FDA’s request,” according to an HHS spokesperson ...

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

Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the ...

Sarepta Therapeutics hired a Trump-connected lobbying firm after the death of a teenage boy treated with its Duchenne ...

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 ...

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 ...

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

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.

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.

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 ...