WebIntroduction: Spinal muscular atrophy (SMA) is a progressive neurological disease with autosomal recessive transmission that affects motor neurons, causing their loss and resulting in muscle waste and motor deficiency. Nusinersen, the first SMN2 pre-mRNA targeted therapy approved by the Food and Drug Administration and the European … WebNusinersen [27] is the first approved SMN2 pre-mRNA targeted therapy for SMA (December 2016 for United States of America and May 2024 for Europe) [28,29]. It consists
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