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 ...

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 ...

Elevidys has fast become Sarepta’s top product. The company reported $820.8 million in Elevidys revenue in 2024, up from $200.3 million in 2023, the year it entered the market.

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

In the U.S., ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in ...

ELEVIDYS is the only approved gene therapy for families and children devastated by Duchenne, a rare, progressive and ultimately fatal disease. We remain committed to working closely with the FDA ...