Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five DMD trials ...

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

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

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

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.

The company plans to file next year for an accelerated clearance of its "exon-skipping" treatment, which would compete with one of Sarepta's medicines.

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

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

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

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The initial signs of Duchenne muscular dystrophy in a 3 or 4-year-old child may be subtle, explains Dr. Alexandra ...

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