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 ...

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

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 ...

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

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

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 ...

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

We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases.

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