Gene Therapy for Duchenne Muscular Dystrophy What is Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disease caused by mutations in the dystrophin gene on the X chromosome. The mutation results in a lack of dystrophin in patients' muscles, a protein that keeps muscle cells intact and, when deficient, leads to progressive muscle degeneration and atrophy. DMD also affects the heart and respiratory...
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