The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

These include gene-based therapies (e.g., replacing a patient's faulty DMD genes with normally functioning ones), cell-based therapies (e.g., replacing dystrophin-deficient muscle cells with stem ...

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech ...

New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. Updated ...

Solid Biosciences’ gene therapy candidate for patients with Duchenne muscular dystrophy (DMD) has elicited a 110% expression of the microdystrophin gene. The US-based company’s stock soared by ...

Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...