Spotlight on … Duchenne muscular dystrophy (DMD) and Spinal muscular atrophy (SMA)

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Duchenne muscular dystrophy (DMD) and Spinal muscular atrophy (SMA) are rare neurodegenerative diseases, which cause progressive, proximal-to-distal muscular weakness leading to loss of ambulation and motor function. Worldwide, they are the leading cause of neuromuscular disorders affecting children, which has led to an active research field to discover new DMD and SMA therapies. Since 2013, […]