articleThe LancetDec 1, 2016Closed access

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

RSRichard S. FinkelCAClaudia A. ChiribogaJVJiri VajsarJWJohn W DayJMJacqueline Montes

Nemours Children's Clinic · Columbia University Irving Medical Center · +4 more institutions

PubMed
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Abstract

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Citation impact

1,011
total citations
FWCI
75.28
Percentile
100%
References
30
Citations per year

Authors

14

Topics & keywords

Topics
Keywords
  • Medicine
  • Spinal muscular atrophy
  • SMN1
  • Tolerability
  • Adverse effect
  • Motor neuron
  • Atrophy
  • Pediatrics
UN Sustainable Development Goals
  • Good health and well-being
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