Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

Nat Neurosci. 2017 Apr;20(4):497-499. doi: 10.1038/nn.4508. Epub 2017 Feb 13.
No abstract available

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Discovery / methods
  • Drug Discovery / trends*
  • Humans
  • Muscular Atrophy, Spinal / diagnosis
  • Muscular Atrophy, Spinal / drug therapy*
  • Muscular Atrophy, Spinal / physiopathology
  • Oligonucleotides / therapeutic use*
  • Oligonucleotides, Antisense / therapeutic use*

Substances

  • Oligonucleotides
  • Oligonucleotides, Antisense
  • nusinersen