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 ...

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

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 ...

Finally, they volunteered Chase, who's now 8, and Dylan, who's now 5, for a study testing an experimental gene therapy for Duchenne muscular dystrophy. Shots - Health News ...

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

As mothers of children with this disease, we have wept helplessly in recent months as friends — fellow members of a club we ...