The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

Sarepta Therapeutics said Friday that the deaths of two patients taking its marketed gene therapy Elevidys® (delandistrogene ...

The FDA said it was putting clinical trials for limb girdle muscular dystrophy on hold due to safety concerns. Elevidys received traditional approval in 2024 for patients age 4 and older with the ...

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

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

Shares of Sarepta Therapeutics Inc. (SRPT) traded 20% higher on Tuesday afternoon after the U.S. Food and Drug Administration ...

Elevidys has fast become Sarepta’s top product. The company reported $820.8 million in Elevidys revenue in 2024, up from $200.3 million in 2023, the year it entered the market.

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene ...

Sarepta Therapeutics’ stock was soaring Tuesday after the FDA recommended lifting the pause on the company’s Duchenne muscular dystrophy drug, but analysts worry it will take time for patients and ...

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) Roche will continue its ...

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

In the U.S., ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in ...