Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

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

Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...

Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...

Sarepta’s Elevidys is back on the market for ambulatory patients with Duchenne muscular dystrophy, Health Secretary Robert F.

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

SRPT's Q2 report, set to arrive Aug. 6, is likely to have Elevidys updates, restructuring plans and sales outlook in sharp investor focus.

The FDA is allowing Sarepta to resume shipments of Elevidys (delandistrogene moxeparvovec) to ambulatory patients with Duchenne muscular dystrophy.

Rare disease and gene therapy stocks, battered in recent months by clinical and commercial setbacks, will likely benefit from ...

As Laura Loomer wields a terrifying amount of power, other far-right influencers are accusing her of being a “plant.” ...

I do not work for Big Pharma. I work in support of President Trump on the outside of the administration,’ Loomer tells The ...

Vinay Prasad, a top regulator at the US Food and Drug Administration, ended his short tenure at the agency after conservative ...