Odevixibat treatment in progressive familial intrahepatic cholestasis: a randomised, placebo-controlled, phase 3 trial

Thompson, RJ; Arnell, H; Artan, R; Baumann, U; Calvo, PL; Czubkowski, P; Dalgic, B; D'Antiga, L; Durmaz, O; Fischler, B; Gonzales, E; Grammatikopoulos, T; Gupte, G; Hardikar, W; Houwen, RHJ; Kamath, BM; Karpen, SJ; Kjems, L; Lacaille, F; Lachaux, A; Lainka, E; Mack, CL; Mattsson, JP; McKiernan, P; Ozen, H; Rajwal, SR; Roquelaure, B; Shagrani, M; Shteyer, E; Soufi, N; Sturm, E; Tessier, ME; Verkade, HJ; Horn, P

Thompson, RJ (通讯作者)，Kings Coll London, Inst Liver Studies, London SE5 9RS, England.

LANCET GASTROENTEROLOGY & HEPATOLOGY, 2022; 7 (9): 830

Abstract

Background Progressive familial intrahepatic cholestasis (PFIC) is a group of inherited paediatric liver diseases resulting from mutations in genes th......

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