Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

Adams, David; González‐Duarte, Alejandra; O’Riordan, William; Yang, Chih‐Chao; Ueda, Mitsuharu; V. Kristen, Arnt; Tournev, Ivailo; Schmidt, Hartmut; Coelho, Teresa; L. Berk, John; Lin, Kon-Ping; Vita, Giuseppe; Attarian, Shahram; Planté‐Bordeneuve, Violaine; M. Mezei, Michelle; M. Campistol, Josep; Buades, Juan; H. Brannagan, Thomas; J. Kim, Byoung; Oh, Jeeyoung; Parman, Yeşim; Sekijima, Yoshiki; N. Hawkins, Philip; D. Solomon, Scott; Polydefkis, Michael; James B. Dyck, P.; J. Gandhi, Pritesh; Goyal, Sunita; Chen, Jihong; Strahs, Andrew; V. Nochur, Saraswathy; T. Sweetser, Marianne; Garg, Pushkal; Vaishnaw, Akshay; Gollob, Jared; B. Suhr, Ole

doi:10.1056/nejmoa1716153

Public

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

Published in New England Journal of Medicine • Jul 4, 2018

Authors:

David Adams

Alejandra González‐Duarte

William O’Riordan

Abstract

BACKGROUND: Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin. METHODS: In this phase 3 trial, we randomly assigned patients with hereditary transthyretin amyloidosis with polyneuropathy, in a 2:1 ratio, to receive intravenous ...

Subject

Transthyretin

RNA interference

Amyloidosis

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