News

The BMJ12m
Duchenne gene therapy halted after three deaths and company u turn
The biotechnology company Sarepta Therapeutics has decided to suspend entirely one of its treatments for Duchenne muscular dystrophy (DMD) just days after it refused a request from the US Food and ...
Roche Pauses Contested Gene Therapy’s Shipments Outside US
Roche Holding AG paused some shipments of the controversial gene therapy Elevidys following a similar action by the embattled ...
MedPage Today1d
Drugmaker Refuses FDA's Request to Pull Gene Therapy Tied to Patient Deaths
The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...
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 ...
Fierce Biotech1d
Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy
The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle ...
MedPage Today on MSN6d
Duchenne Gene Therapy Will Undergo Changes After Patient Deaths
At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...
STAT4dOpinion
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 ...