Key Takeaways Some studies of Sarepta Therapeutics' muscular dystrophy drug, Elevidys, have been halted by European ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

Treatment with gene therapy candidate BB-301 led to improvements in swallowing ability for the first three OPMD patients in a ...

The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent ...

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for ...

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients ...

One of the demands raised during the protests was access to 'Elevidys', a one-time gene therapy treatment that has the ...

Epicrispr has raised $68m in the initial close of its Series B financing round to commence the clinical trial of EPI-321 for ...

The therapy, EPI-321, is intended to treat cases of facioscapulohumeral muscular dystrophy, a genetic neuromuscular disorder.

In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...