Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Abstract
Background Nusinersen is an antisense oligonucleotide drug that modulates pre–messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been developed for the treatment of spinal muscular atrophy (SMA). Methods We conducted a multicenter, double-blind, sham-controlled, phase 3 trial of nusinersen in 126 children with SMA who had symptom onset after 6 months of age. The children were randomly assigned, in a 2:1 ratio, to undergo...
Paper Details
Title
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Published Date
Feb 14, 2018
Volume
378
Issue
7
Pages
625 - 635
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