Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

Adams D., Polydefkis M., Gonzalez-Duarte A., Wixner J., Kristen A., Schmidt H. H., ...More

LANCET NEUROLOGY, vol.20, no.1, pp.49-59, 2021 (SCI-Expanded) identifier identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 20 Issue: 1
  • Publication Date: 2021
  • Doi Number: 10.1016/s1474-4422(20)30368-9
  • Journal Name: LANCET NEUROLOGY
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, CINAHL, EMBASE, MEDLINE, Psycinfo
  • Page Numbers: pp.49-59
  • Istanbul University Affiliated: No

Abstract

Background Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We assessed the safety and efficacy of long-term treatment with patisiran, an RNA interference therapeutic that inhibits TTR production, in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy.