Understanding Duchenne Muscular Dystrophy

One in 3,500 boys in the U.S. have Duchenne muscular dystrophy (DMD), and there are currently no approved disease-modifying treatments. Most children, despite taking steroids to mitigate progressive muscle loss, are wheelchair-bound by ageĀ 12, and median survival is ageĀ 25.

DMD is caused by absence of the dystrophin protein, resulting in abnormal muscle structure and function and buildup of fibrosis in muscle, which diminishes mobility, pulmonary function, and cardiac function. Constant myofiber breakdown results in persistent activation of myofibroblasts and aberrant production of extracellular matrix (ECM) proteins, including collagens and fibronectin, leading to extensive fibrosis in skeletal muscles.

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