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

Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort.

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death of a patient taking Sarepta’s Elevidys raises important safety questions.

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five DMD trials to keep an eye on in 2025.

Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...

WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II ...

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

Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...

The Cambridge-based biotech's experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, ...