Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial

Adams, D; Tournev, IL; Taylor, MS; Coelho, T; Planté-Bordeneuve, V; Berk, JL; González-Duarte, A; Gillmore, JD; Low, SC; Sekijima, Y; Obici, L; Chen, C; Badri, P; Arum, SM; Vest, J; Polydefkis, M

Adams, D (通讯作者)，Univ Paris Saclay, CHU Bicetre, Neurol Dept, INSERM U1195,Natl Reference Ctr FAP NNERF, 78 Rue Gen Leclerc, F-94270 Le Kremlin Bicetre, France.

AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, 2023; 30 (1): 18

Abstract

Background The study objective was to assess the effect of vutrisiran, an RNA interference therapeutic that reduces transthyretin (TTR) production, in......

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