Elevidys was granted expanded approval by the U.S. Food ... Górecki et al, Is dystrophin immunogenicity a barrier to ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

A few weeks after Sarepta Therapeutics confirmed a patient died after receiving its drug Elevidys, research published April 3 ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy, did not achieve its primary endpoint, according to findings ...

CAMBRIDGE, Mass., April 04, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ...

The determination comes after recruitment and dosing in certain trials were paused after a young patient died due to acute ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

One day after the European Medicines Agency requested that three clinical trials of Elevidys be placed on hold after the ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short ... the treatment with gene therapy such as Elevidys (delandistrogene moxeparvovec) aimed at ...