News

EconoTimes8m
FDA Probes Death of 8-Year-Old Linked to Sarepta’s Elevidys Gene Therapy
The U.S. Food and Drug Administration (FDA) is investigating the death of an eight-year-old boy who received Sarepta ...
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 ...
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 ...
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 ...
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 ...
Medscape9h
EMA Says No to Duchenne Gene TherapyElevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
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 ...