Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

Benson, MD; Waddington-Cruz, M; Berk, JL; Polydefkis, M; Dyck, PJ; Wang, AK; Plante-Bordeneuve, V; Barroso, FA; Merlini, G; Obici, L; Scheinberg, M; Brannagan, TH; Litchy, WJ; Whelan, C; Drachman, BM; Adams, D; Heitner, SB; Conceicao, I; Schmidt, HH;

Coelho, T (reprint author), Hosp Santo Antonio, Ctr Hosp Porto, P-4099001 Porto, Portugal.

NEW ENGLAND JOURNAL OF MEDICINE, 2018; 379 (1): 22

Abstract

BACKGROUND Hereditary transthyretin amyloidosis is caused by pathogenic single-nucleotide variants in the gene encoding transthyretin (TTR) that induc......

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