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

This week on "The Readout LOUD" podcast, a mother whose son has Duchenne muscular dystrophy shares her perspective on Sarepta ...

When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The ...

Sarepta stock plunged again Thursday on a report the FDA will require additional clinical testing to validate the safety ...

Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...

The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying the agency was investigating the three deaths due to acute liver failure ...

While Sarepta has now consented to the FDA’s request to stop selling Elevidys, the company’s brief standoff with the agency ...

Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s ...

Second patient death from liver failure after Sarepta's Elevidys gene therapy triggers FDA investigation. Stock crashes 41% ...

Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its ...

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 ...

Gene therapy approach developed offers new hope for those with Duchenne muscular dystrophy by restoring the full-length dystrophin protein. Systemic delivery of full-length dystrophin in Duchenne ...