Abstract:Spinal muscular atrophy is a genetically inherited severe neuromuscular disease due to degeneration of alpha motor neurons in spinal cord and brainstem. SMA is divided into clinical subtypes using the age of onset and achieved development milestones. It is characterized by tetraparesis with a proximal pattern, affecting predominately the lower limbs and hypotonia. Recently, the paradigm of SMA has changed after approval of the first disease-modifying therapy. The pharmacological treatment will have a better ou… Show more
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