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

Adams, David; Polydefkis, Michael; Gonzalez-Duarte, Alejandra; Wixner, Jonas; Kristen, Arnt; Schmidt, Hartmut; Berk, John; Losada Lopez, Ines; Dispenzieri, Angela; Quan, Dianna; Conceicao, Isabel; Slama, Michel; Gillmore, Julian; Kyriakides, Theodoros; Ajroud-Driss, Senda; Waddington-Cruz, Marcia; Mezei, Michelle; Plante-Bordeneuve, Violaine; Attarian, Shahram; Mauricio, Elizabeth; Brannagan, Thomas; Ueda, Mitsuharu; Aldinc, Emre; Wang, Jing; White, Matthew; Vest, John; Berber, Erhan; Sweetser, Marianne; Coelho, Teresa

doi:10.1016/s1474-4422(20)30368-9

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, Scopus)

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.