A proteomic study of the phase 1/2a delandistrogene moxeparvovec trial shows that high-dose AAV gene therapy triggers a sharp ...

A new Pediatric Research reflection by Alex Smith traces nearly twenty years of living alongside his son Harrison's Duchenne ...

Duchenne muscular dystrophy (DMD) is a rare but deadly disease that affects approximately 1 in 5000 boys. DMD causes severe muscle degeneration that eventually leads to heart and lung failure, and ...

Nature Medicine explores the latest translational and clinical research news, with a delayed FDA decision on Sarepta’s adeno-associated virus gene therapy SRP-9001. The US Food and Drug Administration ...

COLUMBUS , Ohio – New research shows that a wasting condition responsible for nearly a third of all cancer deaths involves the loss of an essential muscle protein that is also lost in people with ...

Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the ...

Top row: skeletal muscle without dystrophin. Left to right: skeletal muscle nuclei (blue), skeletal muscle (red), missing dystrophin, overlay of all three images to the left. Bottom row: skeletal ...

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Muscle contraction is the tightening, shortening, or lengthening of muscles when you do some activity. It can happen when you hold or pick up something, or when you stretch or exercise with weights.