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

The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...

Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...

A senior official at the US Food and Drug Administration has suddenly resigned after only three months in the job. Vinay Prasad’s departure followed controversy over a treatment for Duchenne muscular ...

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

Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...

The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

Sarepta will halt all shipments of Elevidys, its drug for Duchenne muscular dystrophy patients, by Tuesday evening.

Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for 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 ...

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 ...