News

Fierce Pharma6h

FDA weighs new study requirements for Sarepta to confirm safety of gene therapy Elevidys: report

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

Sarepta’s Risky Gamble: Gene Therapy Stays on Market Despite FDA Request

When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics. The ...
USA Today2y

Duchenne muscular dystrophy treatments, gene therapy spark hope - USA TODAY

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 ...
Science Daily5y

New gene correction therapy for Duchenne muscular dystrophy

New gene correction therapy for Duchenne muscular dystrophy. ScienceDaily . Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2020 / 01 / 200127134851.htm ...
MedCity News2y

FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy

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 ...
NPR2y

Gene therapy for muscular dystrophy stirs hopes and controversy

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 ...

FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
STAT6dOpinion

We are mothers of Duchenne patients. Recent setbacks with Sarepta must not stop progress

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