Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder that is characterized by atrophy and weakness of the skeletal muscles of the limbs and trunk and of the bulbar and respiratory muscles. It is caused by homozygous deletions or loss-of-function mutations in the gene…

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