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Disease burden and management of Crigler-Najjar syndrome: Report of a world registry.

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作者单位: Department of Gastroenterology & Hepatology and Tytgat Institute for Liver and Intestinal Research, Amsterdam Gastroenterology & Metabolism, Amsterdam University Medical Centers, Amsterdam, the Netherlands. [1] Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany. [2] Laboratoire de Génétique Humaine, Faculté de Médecine de Tunis (Laboratory of Human Genetics, Faculty of Medicine of Tunis, Université de Tunis El Manar (University of Tunis El Manar), Tunis, Tunisia. [3] Service des Maladies Congénitales et Héréditaires (Department of Hereditary and Congenital Disorders), Hôpital Charles Nicolle (Charles Nicolle Hospital), Tunis, Tunisia. [4] Department of Hereditary Diseases of Hepatic Metabolism, Hôpital Antoine Béclère, Clamart, France. [5] Clinic for Special Children, Strasburg, Pennsylvania, USA. [6] Department of Pediatrics, Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia, Pennsylvania, USA. [7] Department of Gastroenterology and Hepatology, Erasmus University Medical Center, Sophia Children's Hospital, Rotterdam, the Netherlands. [8] Department of Pediatric Gastroenterology, Erasmus University Medical Center, Sophia Children's Hospital, Rotterdam, the Netherlands. [9] Department of Translational Medicine, Federico II University of Naples, Naples, Italy. [10] Third Military Medical University, Chongqing, China. [11] Department of Pediatric Gastroenterology, Baskent University Faculty of Medicine, Ankara, Turkey. [12] Metabolomics and Genomics Research Center, Endocrinology and Metabolism Molecular-Cellular Sciences Institute, Tehran University of Medical Sciences, Tehran, Iran. [13] Health research Institute, Research Center of Thalassemia & Hemoglobinopathy, Ahvaz Jundishapur University of Medical Sciences, Ahvaz, Iran. [14] Department of Pediatrics and Pediatric Hepatology, Faculty of Medicine, Cairo University, Cairo, Egypt. [15] Paediatric Gastroenterology and Nutrition, Shaare Zedek Medical Center, Jerusalem, Israel. [16] Department of Paediatric Gastroenterology, Azienda Ospedaliera Papa Giovanni XXIII, Bergamo, Italy. [17] Spark Therapeutics, Philadelphia, Pennsylvania, USA. [18] Telethon Institute of Genetics and Medicine, Pozzuoli, Italy. [19] Departments of Pediatrics and Molecular, Cell & Cancer Biology, University of Massachusetts School of Medicine, Worcester, Massachusetts, USA. [20]
DOI 10.1111/liv.15239
PMID 35274801
发布时间 2022-07-12
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Liver international : official journal of the International Association for the Study of the Liver

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