Jefferies analysts said these detailed safety outcomes confirm the gene therapy's positive risk/benefit profile in ambulatory ...

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

After a second Elevidys patient death from acute liver failure, Sarepta in July launched a major restructuring that involves ...

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 ...

U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a ...

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

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

Vinay Prasad has stepped down as head of the FDA’s biologics division just two months into the job, following growing ...

Reports of troubles linked to Sarepta’s Elevidys treatment for Duchenne muscular dystrophy had been popping up on social media for months — beyond the two deaths the company had already ...

Cayse, 5, who has Duchenne muscular dystrophy, was supposed to be dosed with a gene therapy this summer before the FDA asked that it be pulled from the market. Photo courtesy of Jennifer Hill Blair ...

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

The Trump FDA tries to kill a therapy that has helped boys with a deadly diagnosis.