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

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 ...

Dec. 16, 2024 — A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an ...

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

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

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 ...

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.

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.

Pfizer is “evaluating appropriate next steps” for its Duchenne muscular dystrophy (DMD) gene therapy after it failed a phase 3 trial. Fordadistrogene movaparvovec (PF-06939926) was unable to ...