The death of a 16-year-old boy taking a gene therapy this month dealt a fresh blow to the Duchenne muscular dystrophy ...

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

PTC Therapeutics said on Friday its inherited progressive muscle-wasting disorder drug will no longer be available for sale in the EU as the European Commission has decided not to renew its marketing ...

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

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

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Avidity Biosciences, Inc.'s innovative AOC platform and promising clinical programs make it a high-risk, high-reward ...

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

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...

Encelto is small, semi-permeable capsule implanted in the eye that contains allogeneic retinal pigment epithelium cells ...

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...