Bettina E Hansen, Shannon M Vandriel, Pamela Vig, Will Garner, Douglas B Mogul, Kathleen M Loomes, David A Piccoli, Elizabeth B Rand, Irena Jankowska, Piotr Czubkowski, Dorota Gliwicz-Miedzińska, Emmanuel M Gonzales, Emmanuel Jacquemin, Jérôme Bouligand, Lorenzo D'Antiga, Emanuele Nicastro, Henrik Arnell, Björn Fischler, Étienne Sokal, Tanguy Demaret, Susan Siew, Michael Stormon, Saul J Karpen, Rene Romero, Noelle H Ebel, Jeffrey A Feinstein, Amin J Roberts, Helen M Evans, Shikha S Sundaram, Alexander Chaidez, Winita Hardikar, Sahana Shankar, Ryan T Fischer, Florence Lacaille, Dominique Debray, Henry C Lin, M Kyle Jensen, Catalina Jaramillo, Palaniswamy Karthikeyan, Giuseppe Indolfi, Henkjan J Verkade, Catherine Larson-Nath, Ruben E Quiros-Tejeira, Pamela L Valentino, Maria Rogalidou, Antal Dezsőfi, James E Squires, Kathleen Schwarz, Pier Luigi Calvo, Jesus Quintero Bernabeu, Andréanne N Zizzo, Gabriella Nebbia, Pinar Bulut, Ermelinda Santos-Silva, Rima Fawaz, Silvia Nastasio, Wikrom Karnsakul, María Legarda Tamara, Cristina Molera Busoms, Deirdre Kelly, Thomas Damgaard Sandahl, Carolina Jimenez-Rivera, Jesus M Banales, Quais Mujawar, Li-Ting Li, Huiyu She, Jian-She Wang, Kyung Mo Kim, Seak Hee Oh, Maria Camila Sanchez, Maria Lorena Cavalieri, Way Seah Lee, Christina Hajinicolaou, Chatmanee Lertudomphonwanit, Orith Waisbourd-Zinman, Cigdem Arikan, Seema Alam, Elisa Carvalho, Melina Melere, John Eshun, Zerrin Önal, Dev M Desai, Sabina Wiecek, Raquel Borges Pinto, Victorien M Wolters, Jennifer Garcia, Marisa Beretta, Nanda Kerkar, Jernej Brecelj, Nathalie Rock, Eberhard Lurz, Niviann Blondet, Uzma Shah, Richard J Thompson, Binita M Kamath
BACKGROUND AND AIMS: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is the first-approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the Global ALagille Alliance (GALA) study...
December 25, 2023: Hepatology: Official Journal of the American Association for the Study of Liver Diseases