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

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 ...

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, also known as DMD, is a genetic, progressive condition that causes loss of muscle function over time. It’s mostly seen in boys and men, but it can also occur, often ...

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

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 ...

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

Cardiomyopathy is the leading cause of death in patients with Duchenne muscular dystrophy, with roughly 90% developing a dilated cardiomyopathy by age 18. 2,3 Cardiomyocyte degeneration results in ...

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.