Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is characterized by progressive muscle atrophy and weakness, with an estimated incidence of 1 in 11,000 live births. Approximately 60% of infants who are born with spinal muscular atrophy have type 1 disease, which is…

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