Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its ...

The FDA expanded the approval of delandistrogene moxeparvovec-rokl (Elevidys) gene therapy for Duchenne muscular dystrophy on Thursday to include ambulatory or non-ambulatory patients ages 4 ...

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the ...

The first gene therapy that can treat Duchenne muscular dystrophy (DMD) has been approved by the U.S. Food and Drug Administration; it will be marketed as Elevidys (delandistrogene ...

The Duchenne muscular dystrophy drugs now available only treat the symptoms of the rare muscle-wasting disorder. On Thursday, the FDA approved a Sarepta Therapeutics gene therapy, making it ...

New gene correction therapy for Duchenne muscular dystrophy. ScienceDaily . Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2020 / 01 / 200127134851.htm ...