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Artigo Produção Nacional Revisado por pares
BIBTEX RIS

Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

2024; Elsevier BV; Volume: 9; Issue: 7 Linguagem: Inglês

10.1016/s2468-1253(24)00080-3

ISSN

2468-1253

Autores

Alexander Miethke, Adib Moukarzel, Gilda Porta, Joshue Covarrubias Esquer, Piotr Czubkowski, Felipe Ordonez, Antonella Mosca, Amal Aqul, Robert H. Squires, Étienne Sokal, Daniel D’Agostino, Ulrich Baumann, Lorenzo D’Antiga, Nagraj Kasi, Nolwenn Laborde, Çiğdem Arıkan, Chuan-Hao Lin, Susan Gilmour, Naveen Mittal, Fang Kuan Chiou, Simon Horslen, Wolf‐Dietrich Huber, Thomas Jaecklin, Tiago Nunes, Anamaria Lascau, Lara Longpre, Douglas Mogul, Will Garner, Pamela Vig, Vera Hupertz, Regino P. González‐Peralta, Udeme D. Ekong, Jane Hartley, Noémie Laverdure, Nadia Ovchinsky, Richard J. Thompson,

Tópico(s)

Liver Diseases and Immunity

Resumo

Progressive familial intrahepatic cholestasis (PFIC) is a group of autosomal recessive disorders, the most prevalent being BSEP deficiency, resulting in disrupted bile formation, cholestasis, and pruritus. Building on a previous phase 2 study, we aimed to evaluate the efficacy and safety of maralixibat-an ileal bile acid transporter inhibitor-in participants with all types of PFIC.

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