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

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

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines ...

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 ...

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.

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

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients ...

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

For millions of people, losing muscle isn't just about weakness; it's about losing independence. Whether caused by Duchenne ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.