Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

Adams, D; Gonzalez-Duarte, A; O'Riordan, WD; Yang, CC; Ueda, M; Kristen, AV; Tournev, I; Schmidt, HH; Coelho, T; Berk, JL; Lin, KP; Vita, G; Attarian, S; Plante-Bordeneuve, V; Mezei, MM; Campistol, JM; Buades, J; Brannagan, TH; Kim, BJ; Oh, J; Parman

Adams, D (reprint author), CHU Bicetre, Dept Neurol, 78 Rue Gen Leclerc, F-94275 Le Kremlin Bicetre, France.

NEW ENGLAND JOURNAL OF MEDICINE, 2018; 379 (1): 11

Abstract

BACKGROUND Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin. METHODS In this......

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