Effectiveness and Safety of Nusinersen and Risdiplam in Spinal Muscular Atrophy: A Systematic Review.
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DOI: 10.1002/acn3.70274
Abstract: OBJECTIVE Spinal Muscular Atrophy (SMA) is a rare genetic disorder marked by progressive muscle weakness and mobility loss. It has a profound physical, emotional and social impact on patients and caregivers, requiring comprehensive medical and… read more here.
Keywords: spinal muscular; nusinersen risdiplam; review; muscular atrophy ... See more keywords