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Papers of the Week

2020 Apr

J Neurol



Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis.


Coelho T, Yarlas A, Waddington-Cruz M, White MK, Sikora Kessler A, Lovley A, Pollock M, Guthrie S, Ackermann EJ, Hughes SG, Karam C, Khella S, Gertz M, Merlini G, Obici L, Schmidt HH, Polydefkis M, Dyck JPB, Brannagan Iii TH, Conceição I, et al.
J Neurol. 2020 Apr; 267(4):1070-1079.
PMID: 31853709.


To examine the impact on quality of life (QOL) of patients with hATTR amyloidosis with polyneuropathy treated with inotersen (Tegsedi™) versus placebo.