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 ...

With a strong launch underway for a bladder cancer gene therapy, Ferring Pharmaceuticals is finding the kind of commercial ...

CBER Chief Vinay Prasad reclaimed his job less than two weeks after his mysterious exit; MAHA implementor Gray Delany is out ...

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

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 ...

Vinay Prasad is once again head of the FDA’s Center for Biologics Evaluation and Research, after Laura Loomer led a campaign ...

Charlie has Duchenne muscular dystrophy, a degenerative genetic disease that, until recently, has guaranteed death by early adulthood from cardiac or respiratory failure.

A Food and Drug Administration official who stepped down less than two weeks ago is getting his job back as the agency’s top ...

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.

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.

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 ...