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 ...
Sarepta Therapeutics crisis is huge blow to Duchenne families, company
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
STAT2dOpinion
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 ...
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 ...
MedPage Today1y
Gene Therapy for Duchenne Muscular Dystrophy Gets Expanded Approval
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 ...
People9mon
Boy with Duchenne's Gets Gene Therapy 3 Days Before He’s Ineligible ...
Melanie Sanford fought to get her son Hudson a breakthrough gene therapy to stop the progression of the fatal disease Duchenne Muscular Dystrophy. She rushed to get him access just days ...
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 ...
Muscular Dystrophy News3d
Dosing begins in gene therapy trial for DMD-linked heart disease
The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated ...