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Nusinersen Versus Sham Control in Later-Onset Spinal Muscular Atrophy NEJM 378:625-635, Mercuri, E.,et al, 2018 See this aricle in Pubmed Article Abstract Among children with later-onset SMA, those who received nusinersen had significant and clinically meaningful improvement in motor function as compared with those in the control group.

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(click to filter results - removes previous filter) children efficacy Kugelberg-Welander syndrome mortality motor neuron disease nusinersen safety spinal muscular atrophy spinal muscular atrophy,intermediate form treatment of neurologic disorder

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