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EMJ Neurology 8.1 2020
July 2020
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18 Mins
27
Jan
New Advances in the Treatment of Duchenne Muscular Dystrophy and Spinal Muscular Atrophy
Both spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) are monogenic neuromuscular diseases, which cause progressive proximal-to-distal muscular weakness, leading to loss of motor function and related pulmonary and musculoskeletal co-morbidities and reduced survival.