Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

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

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

When her son reached three last year, a new name loomed over their lives — Duchenne Muscular Dystrophy (DMD), a ...

Parents Jamie, 33, and Bobbie, 32, have spent the last three years desperately seeking treatments abroad and have been ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for ...

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

Little Jamie Tierney Jr's parents have been desperately trying to save him since he was diagnosed in March 2022.

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

One of the demands raised during the protests was access to 'Elevidys', a one-time gene therapy treatment that has the ...

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