This symposium review of presentations made at the World Muscle Society (WMS) 2021 Virtual Congress draws a spotlight upon two monogenic neuromuscular diseases: spinal muscular atrophy (SMA) and Duchenne muscular dystrophy.
EMJ Neurology 10.1 2022
January 2022
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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.