Order byBest matchMost fresh
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 Sarepta Therapeutics' muscular disorder gene therapy Elevidys. The death occurred on June 7,
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 individuals with Duchenne muscular dystrophy (DMD) Roche will continue its dialogue with the EMA to explore a potential path forward to make Elevidys available to individuals living with DMD in the EURoche believes the benefit-risk remains positive in the ambulatory Duchenne populationElevidys is the first and only disease-modifying gene therapy for DMD Basel,
STAT1d
For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are caught in the middle.
FDA Probes Death of Boy in Brazil on Sarepta’s Elevidys
US regulators are investigating the death of an 8-year-old boy in Brazil who received Sarepta Therapeutics Inc.’s Elevidys.
BioPharma Dive1d
Sarepta woes mount as Duchenne gene therapy knocked back in Europe
The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could further jeopardize Sarepta’s financial future.
BioSpace1d
Sarepta Fallout Continues With EU’s Negative Opinion of Elevidys in Ambulatory Patients
The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne muscular dystrophy.
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 gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company's treatments.
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 declining and that it was getting worse and worse," Stephanie said. Pediatric neurologists at Nemours Children's Hospital found a way to give Logan more time to be a kid.
Feedback