Alagille Syndrome: Diagnostic Challenges and Advances in Management by Mohammed D. Ayoub, Binita M. Kamath

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Severe Neonatal Cholestasis as an Early Presentation of McCune- Albright Syndrome by Nicole Coles, Ian Comeau, Tatiana Munoz, Jennifer Harrington, Roberto Mendoza-Londono, Andreas Schulze, Sari Kives, Binita M. Kamath, Jill Hamilton

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Magnetic Resonance Cholangiopancreatography Severity Predicts Disease Outcomes in Pediatric Primary Sclerosing Cholangitis: A Reliability and Validity Study by Kedar Patil, Amanda Ricciuto, Alaa Alsharief, Jehan Al‐Rayahi, Afsaneh Amirabadi, Peter C. Church, Binita M. Kamath, Mary‐Louise C. Greer

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Loss of Mtm1 causes cholestatic liver disease in a model of X-linked myotubular myopathy by Sophie Karolczak, Ashish R. Deshwar, Evangelina Aristegui, Binita M. Kamath, Michael W. Lawlor, Gaia Andreoletti, Jonathan Volpatti, Jillian L. Ellis, Chunyue Yin, James J. Dowling

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Loss of zebrafish pkd1l1 causes biliary defects that have implications for biliary atresia splenic malformation by Rouknuddin Q. Ali, Anne Meyer-Miner, Marie David-Rachel, Fiona J. H. Lee, Benjamin J. Wilkins, Saul J. Karpen, Brian Ciruna, Anand Ghanekar, Binita M. Kamath

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Findings in percutaneous trans-hepatic cholecysto-cholangiography in neonates and infants presenting with conjugated hyperbilirubinemia: emphasis on differential diagnosis and chol... by Dimitri A. Parra, Shannon E. Peters, Ruhail Kohli, Racha Chamlati, Bairbre L. Connolly, Justyna M. Wolinska, Vicky L. Ng, Michael J. Temple, Philip R. John, Binita M. Kamath, Simon C. Ling, Annie Fecteau, Afsaneh Amirabadi, Joao G. Amaral

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Use of funded multicenter prospective longitudinal databases to inform clinical trials in rare diseases—Examination of cholestatic liver disease in Alagille syndrome by Benjamin L. Shneider, Binita M. Kamath, John C. Magee, Nathan P. Goodrich, Kathleen M. Loomes, Wen Ye, Cathie Spino, Estella M. Alonso, Jean P. Molleston, Jorge A. Bezerra, Kasper S. Wang, Saul J. Karpen, Simon P. Horslen, Stephen L. Guthery, Philip Rosenthal, Robert H. Squires, Ronald J. Sokol, for the Childhood Liver Disease Research Network (ChiLDReN)

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Placebo‐Controlled Randomized Trial of an Intestinal Bile Salt Transport Inhibitor for Pruritus in Alagille Syndrome by Benjamin L. Shneider, Cathie Spino, Binita M. Kamath, John C. Magee, Lee M. Bass, Kenneth D. Setchell, Alexander Miethke, Jean P. Molleston, Cara L. Mack, Robert H. Squires, Karen F. Murray, Kathleen M. Loomes, Philip Rosenthal, Saul J. Karpen, Daniel H. Leung, Stephen L. Guthery, Danny Thomas, Averell H. Sherker, Ronald J. Sokol, for the Childhood Liver Disease Research Network

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Unraveling the Relationship Between Itching, Scratch Scales, and Biomarkers in Children With Alagille Syndrome by Binita M. Kamath, Cathie Spino, Richard McLain, John C. Magee, Emily M. Fredericks, Kenneth D. Setchell, Alexander Miethke, Jean P. Molleston, Cara L. Mack, Robert H. Squires, Estella M. Alonso, Karen F. Murray, Kathleen M. Loomes, M. Kyle Jensen, Saul J. Karpen, Philip Rosenthal, Danny Thomas, Ronald J. Sokol, Benjamin L. Shneider, for the Childhood Liver Disease Research Network (ChiLDReN)

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Initial assessment of the infant with neonatal cholestasis-Is this biliary atresia? by Benjamin L Shneider, Jeff Moore, Nanda Kerkar, John C Magee, Wen Ye, Saul J Karpen, Binita M Kamath, Jean P Molleston, Jorge A Bezerra, Karen F Murray, Kathleen M Loomes, Peter F Whitington, Philip Rosenthal, Robert H Squires, Stephen L Guthery, Ronen Arnon, Kathleen B Schwarz, Yumirle P Turmelle, Averell H Sherker, Ronald J Sokol, Childhood Liver Disease Research Network

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Impact of long‐term administration of maralixibat on children with cholestasis secondary to Alagille syndrome by Benjamin L. Shneider, Catherine A. Spino, Binita M. Kamath, John C. Magee, Rosalinda V. Ignacio, Suiyuan Huang, Simon P. Horslen, Jean P. Molleston, Alexander G. Miethke, Rohit Kohli, Daniel H. Leung, M. Kyle Jensen, Kathleen M. Loomes, Saul J. Karpen, Cara Mack, Philip Rosenthal, Robert H. Squires, Alastair Baker, Sanjay Rajwal, Deirdre Kelly, Ronald J. Sokol, Richard J. Thompson, for ChiLDReN and UK IMAGO/IMAGINE Investigators

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Outcomes of Childhood Cholestasis in Alagille Syndrome: Results of a Multicenter Observational Study by Binita M. Kamath, Wen Ye, Nathan P. Goodrich, Kathleen M. Loomes, Rene Romero, James E. Heubi, Daniel H. Leung, Nancy B. Spinner, David A. Piccoli, Estella M. Alonso, Stephen L. Guthery, Saul J. Karpen, Cara L. Mack, Jean P. Molleston, Karen F. Murray, Philip Rosenthal, James E. Squires, Jeffrey Teckman, Kasper S. Wang, Richard Thompson, John C. Magee, Ronald J. Sokol, for the Childhood Liver Disease Research Network (ChiLDReN)

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Nonfasted Liver Stiffness Correlates with Liver Disease Parameters and Portal Hypertension in Pediatric Cholestatic Liver Disease by Benjamin L. Shneider, Nathan P. Goodrich, Wen Ye, Cindy Sawyers, Jean P. Molleston, Robert M. Merion, Daniel H. Leung, Saul J. Karpen, Binita M. Kamath, Laurel Cavallo, Kasper Wang, Jeffrey H. Teckman, James E. Squires, Shikha S. Sundaram, Philip Rosenthal, Rene Romero, Karen F. Murray, Kathleen M. Loomes, M. Kyle Jensen, Jorge A. Bezerra, Lee M. Bass, Ronald J. Sokol, John C. Magee, For the Childhood Liver Disease Research Network (ChiLDReN)

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Precision Health Resource of Control iPSC Lines for Versatile Multilineage Differentiation by Matthew R. Hildebrandt, Miriam S. Reuter, Wei Wei, Naeimeh Tayebi, Jiajie Liu, Sazia Sharmin, Jaap Mulder, L. Stephen Lesperance, Patrick M. Brauer, Rebecca S.F. Mok, Caroline Kinnear, Alina Piekna, Asli Romm, Jennifer Howe, Peter Pasceri, Guoliang Meng, Matthew Rozycki, Deivid C. Rodrigues, Elisa C. Martinez, Michael J. Szego, Juan C. Zúñiga-Pflücker, Michele K. Anderson, Steven A. Prescott, Norman D. Rosenblum, Binita M. Kamath, Seema Mital, Stephen W. Scherer, James Ellis

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Interim results from an ongoing, open-label, single-arm trial of odevixibat in progressive familial intrahepatic cholestasis by Richard J. Thompson, Reha Artan, Ulrich Baumann, Pier Luigi Calvo, Piotr Czubkowski, Buket Dalgic, Lorenzo D’Antiga, Angelo Di Giorgio, Özlem Durmaz, Emmanuel Gonzalès, Tassos Grammatikopoulos, Girish Gupte, Winita Hardikar, Roderick H.J. Houwen, Binita M. Kamath, Saul J. Karpen, Florence Lacaille, Alain Lachaux, Elke Lainka, Kathleen M. Loomes, Cara L. Mack, Jan P. Mattsson, Patrick McKiernan, Quanhong Ni, Hasan Özen, Sanjay R. Rajwal, Bertrand Roquelaure, Eyal Shteyer, Etienne Sokal, Ronald J. Sokol, Nisreen Soufi, Ekkehard Sturm, Mary Elizabeth Tessier, Wendy L. van der Woerd, Henkjan J. Verkade, Jennifer M. Vittorio, Terese Wallefors, Natalie Warholic, Qifeng Yu, Patrick Horn, Lise Kjems

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Gamma Glutamyltransferase Reduction Is Associated With Favorable Outcomes in Pediatric Primary Sclerosing Cholangitis by Mark R. Deneau, Cara Mack, Reham Abdou, Mansi Amin, Achiya Amir, Marcus Auth, Fateh Bazerbachi, Anne Marie Broderick, Albert Chan, Matthew DiGuglielmo, Wael El‐Matary, Mounif El‐Youssef, Federica Ferrari, Katryn N. Furuya, Frederic Gottrand, Nitika Gupta, Matjaž Homan, M.K. Jensen, Binita M. Kamath, Kyung Mo Kim, Kaija‐Leena Kolho, Anastasia Konidari, Bart Koot, Raffaele Iorio, Mercedes Martinez, Parvathi Mohan, Sirish Palle, Alexandra Papadopoulou, Amanda Ricciuto, Lawrence Saubermann, Pushpa Sathya, Eyal Shteyer, Vratislav Smolka, Atsushi Tanaka, Pamela L. Valentino, Raghu Varier, Veena Venkat, Bernadette Vitola, Miriam B. Vos, Marek Woynarowski, Jason Yap, Tamir Miloh

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Genotype-phenotype relationships of truncating mutations, p.E297G and p.D482G in bile salt export pump deficiency by Antonia Felzen, Daan B.E. van Wessel, Emmanuel Gonzales, Richard J. Thompson, Irena Jankowska, Benjamin L. Shneider, Etienne Sokal, Tassos Grammatikopoulos, Agustina Kadaristiana, Emmanuel Jacquemin, Anne Spraul, Patryk Lipiński, Piotr Czubkowski, Nathalie Rock, Mohammad Shagrani, Dieter Broering, Emanuele Nicastro, Deirdre Kelly, Gabriella Nebbia, Henrik Arnell, Björn Fischler, Jan B.F. Hulscher, Daniele Serranti, Cigdem Arikan, Esra Polat, Dominique Debray, Florence Lacaille, Cristina Goncalves, Loreto Hierro, Gema Muñoz Bartolo, Yael Mozer-Glassberg, Amer Azaz, Jernej Brecelj, Antal Dezsőfi, Pier Luigi Calvo, Enke Grabhorn, Steffen Hartleif, Wendy J. van der Woerd, Binita M. Kamath, Jian-She Wang, Liting Li, Özlem Durmaz, Nanda Kerkar, Marianne Hørby Jørgensen, Ryan Fischer, Carolina Jimenez-Rivera, Seema Alam, Mara Cananzi, Noemie Laverdure, Cristina Targa Ferreira, Felipe Ordoñez Guerrero, Heng Wang, Valerie Sency, Kyung Mo Kim, Huey-Ling Chen, Elisa de Carvalho, Alexandre Fabre, Jesus Quintero Bernabeu, Aglaia Zellos, Estella M. Alonso, Ronald J. Sokol, Frederick J. Suchy, Kathleen M. Loomes, Patrick J. McKiernan, Philip Rosenthal, Yumirle Turmelle, Simon Horslen, Kathleen Schwarz, Jorge A. Bezerra, Kasper Wang, Bettina E. Hansen, Henkjan J. Verkade

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