Outcomes of 38 patients with PFIC3: Impact of genotype and of response to ursodeoxycholic acid therapy

Background & Aims: Progressive familial intrahepatic cholestasis type 3 (PFIC3) is a rare liver disease caused by biallelic variations in ABCB4. Data reporting on the impact of genotype and of response to ursodeoxycholic acid (UDCA) therapy on long-term outcomes are scarce. Methods: We retro...

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Main Authors: Emmanuel Gonzales, Antoine Gardin, Marion Almes, Amaria Darmellah-Remil, Hanh Seguin, Charlotte Mussini, Stéphanie Franchi-Abella, Mathieu Duché, Oanez Ackermann, Alice Thébaut, Dalila Habes, Bogdan Hermeziu, Martine Lapalus, Thomas Falguières, Jean-Philippe Combal, Bernard Benichou, Sonia Valero, Anne Davit-Spraul, Emmanuel Jacquemin
Format: Article
Language:English
Published: Elsevier 2023-10-01
Series:JHEP Reports
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2589555923001751