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

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

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

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 ...

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

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

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

Vinay Prasad has stepped down as head of the FDA’s biologics division just two months into the job, following growing backlash over how he handled Sarepta’s Elevidys, the only approved gene therapy ...

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

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

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

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