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Interim Efficacy and Safety Results from the Phase 2 NURTURE Study Evaluating Nusinersen in Presymptomatic Infants with Spinal Muscular Atrophy (S46.003)
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2017
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Single-arm StudySpinal Cord InjurySpinal DisorderNeurological DisorderMedicinePediatricsProgressive Supranuclear PalsyRehabilitationNeurologySpinal Muscular AtrophyOngoing Phase 2Phase 2Pediatric SpineNeuromuscular PathologyInterim EfficacyNeuromusculoskeletal DisorderIntrathecal Nusinersen
NURTURE (NCT02386553) is an ongoing phase 2, open-label, single-arm study, evaluating intrathecal nusinersen (12-mg equivalent dose) in infants with presymptomatic spinal muscular atrophy (SMA).