DMD – ANIMAL MODELS & PRECLINICAL TREATMENT

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Many serious inherited myopathies are marked by an exaggerated injury response to with muscle contraction, including Duchenne, Becker and several of the limb girdle muscular dystrophies. Amongst these, breakdown in the context of Duchenne muscular dystrophy (DMD) is best understood. Skeletal muscle contraction leads to membrane stress, local calcium influx and muscle fiber breakdown through excessive contractures and activation of apoptotic pathways. We have identified a novel small molecule, EDG-5506 that stabilizes skeletal muscle fibers so that muscle use, either in the context of isolated contraction or exercise, is uncoupled from an exaggerated injury response.

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