Odevixibat therapy in progressive familial intrahepatic cholestasis with MYO5B variants: a retrospective case series

被引：0

作者：

Bertrand Roquelaure ^{[1
]}

Marco Sciveres ^{[2
]}

Tassos Grammatikopoulos ^{[3
]}

Eberhard Lurz ^{[4
]}

Folke Freudenberg ^{[5
]}

Dalila Habes ^{[6
]}

Lionel Thevathasan ^{[7
]}

Fatine Elaraki ^{[8
]}

Emmanuel Gonzales ^{[9
]}

机构：

[1] APHM,Service de Pédiatrie Multidisciplinaire

[2] Hôpital de la Timone Enfants,Pediatric Hepatology and Pediatric Liver Transplantation

[3] ISMETT,Liver Unit and Liver Transplant Program

[4] University of Pittsburgh Medical Center Italy,Institute of Liver Studies

[5] Bambino Gesù Children Hospital,Paediatric Liver, GI and Nutrition Centre and Mowat Labs

[6] King’s College London,Division of Paediatric Gastroenterology and Hepatology, Dr. von Hauner Children’s Hospital

[7] King’s College Hospital NHS Trust,Hépatologie et Transplantation Hépatique Pédiatriques, Centre de Référence de l’Atrésie des Voies Biliaires et des Cholestases Génétiques, AP

[8] University Hospital Munich,HP

[9] LMU,undefined

[10] Klinikum Dritter Orden,undefined

[11] Division of Pediatric Gastroenterology and Hepatology,undefined

[12] FSMR FILFOIE,undefined

[13] ERN RARE LIVER,undefined

[14] Hôpital Bicêtre,undefined

[15] Université Paris-Saclay,undefined

[16] Inserm U 1193,undefined

[17] LT Associates Ltd,undefined

[18] Ipsen,undefined

来源：

Orphanet Journal of Rare Diseases | / 20卷 / 1期

关键词：

Progressive Familial intrahepatic cholestasis; Myosin 5B; Pruritus; Bilirubin; Odevixibat; Serum bile acid; Ileal bile acid transporter inhibitor;

D O I：

10.1186/s13023-025-03728-x

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