Abstract

Nusinersen in spinal muscular atrophy
Original languageEnglish
Pages (from-to)1723-1732-1732
JournalTHE NEW ENGLAND JOURNAL OF MEDICINE
Volume377
DOIs
Publication statusPublished - 2017

Keywords

  • Age of Onset
  • Disease-Free Survival
  • Double-Blind Method
  • Female
  • Humans
  • Infant
  • Injections, Spinal
  • Male
  • Motor Skills
  • Oligonucleotides
  • Oligonucleotides, Antisense
  • RNA, Messenger
  • Respiration, Artificial
  • Spinal Muscular Atrophies of Childhood
  • Survival Analysis
  • Survival of Motor Neuron 2 Protein

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