Sarepta Therapeutics has provided additional safety data on its gene therapy Elevidys as a Duchenne muscular dystrophy ...

Still others wrote letters to the Food and Drug Administration directly. They demanded the FDA restore access to the drug ...

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

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

Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy ...

Like the first death reported in March, Sarepta Therapeutics attributed the fatality to a case of acute liver failure following dosing of the Duchenne muscular dystrophy gene therapy, Elevidys.

The biotech, which cut jobs following the rejection in May of its Barth syndrome therapy, claimed it has addressed the agency ...

Hansa Biosciences’ Idefirix (imlifidase) has reduced patients’ antibodies to a level that allows dosing in patients with ...

U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a muscular dystrophy patient who received a different, experimental treatment died ...

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

New York, June 22, 2023 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene ...