News

MIT Technology Review13h
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.
STAT11h
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 ...
FDA Investigates Death of Boy Who Got Sarepta’s Gene Therapy
US regulators said they are investigating the death of an 8-year-old boy who received a gene therapy from Sarepta ...
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 ...
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 ...
Medscape7h
EMA Says No to Duchenne Gene TherapyElevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
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 ...