Spinraza-a rare disease success story

Gene Ther. 2017 Sep;24(9):497. doi: 10.1038/gt.2017.59.
No abstract available

Publication types

  • Editorial

MeSH terms

  • Genetic Therapy / methods
  • Humans
  • Muscular Atrophy, Spinal / drug therapy*
  • Oligonucleotides / pharmacology*
  • Rare Diseases

Substances

  • Oligonucleotides
  • nusinersen