News

MIT Technology Review1d
The deadly saga of the controversial gene therapy Elevidys
The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.
Two boys died after a gene therapy. This family won’t give up hope.
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.
STAT1d
Europe moves to reject embattled Duchenne muscular dystrophy gene therapy
European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...
Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU
EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...
Sarepta fails to win EU backing for muscle disorder gene therapy
Sarepta Therapeutics failed to win the European drug regulator's backing for its muscle disorder gene therapy on Friday, as ...
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 Biotech4d
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 ...
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 ...