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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