News

STAT13h
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 ...
MIT Technology Review16h
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.
FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy
The patient with Duchenne muscular dystrophy, who died in Brazil, was treated with Elevidys but was not a participant in a ...
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 ...
Health Beat: Duchenne muscular dystrophy
Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...
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 Pharma1d
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 ...