Gene-silencing technology gets first drug approval after 20-year wait

Nature. 2018 Aug;560(7718):291-292. doi: 10.1038/d41586-018-05867-7.
No abstract available

Keywords: Drug discovery; Gene therapy; Medical research.

Publication types

  • News

MeSH terms

  • Amyloid Neuropathies, Familial / genetics
  • Amyloid Neuropathies, Familial / therapy
  • Drug Approval / legislation & jurisprudence*
  • Gene Silencing
  • Humans
  • RNA, Small Interfering* / therapeutic use
  • RNAi Therapeutics / legislation & jurisprudence*
  • RNAi Therapeutics / trends
  • Time Factors
  • United States
  • United States Food and Drug Administration / legislation & jurisprudence

Substances

  • RNA, Small Interfering
  • patisiran

Supplementary concepts

  • Amyloidosis, Hereditary, Transthyretin-Related