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 ...
FDA probes death of patient on Sarepta's Elevidys, partner Roche says death unrelated to therapy
The U.S. Food and Drug Administration said on Friday it was investigating the death of an eight-year-old boy who received ...
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 ...
MIT Technology Review15h
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.
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 ...
Gene therapy maker Sarepta tells FDA it won’t halt shipments despite patient deaths
Drugmaker Sarepta Therapeutics says it won’t comply with a request from U.S. regulators to halt all shipments of its gene ...
Medscape9h
EMA Says No to Duchenne Gene TherapyElevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.