New England Journal of Medicine· 2017Q1· Clinical trial
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
- 2,299citations
- Q1SCImago
- 2017year
Short summary
In infants with spinal muscular atrophy, nusinersen treatment significantly improved motor function and survival compared to a sham control, with 51% achieving motor milestones versus 0% in the control group.
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GeneticsMedicine