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

Author:

Aronson Sem J.1ORCID,Junge Norman2ORCID,Trabelsi Mediha34,Kelmemi Wided3,Hubert Aurelie5,Brigatti Karlla W.6,Fox Michael D.67,de Knegt Robert J.8ORCID,Escher Johanna C.9,Ginocchio Virginia M.10,Iorio Raffaele10ORCID,Zhu Yan11,Özçay Figen12,Rahim Fakher1314ORCID,El‐Shabrawi Mortada H.F.15ORCID,Shteyer Eyal16ORCID,Di Giorgio Angelo17ORCID,D'Antiga Lorenzo17ORCID,Mingozzi Federico18ORCID,Brunetti‐Pierri Nicola1019ORCID,Strauss Kevin A.620ORCID,Labrune Philippe5,Mrad Ridha34,Baumann Ulrich2,Beuers Ulrich1ORCID,Bosma Piter J.1,

Affiliation:

1. Department of Gastroenterology & Hepatology and Tytgat Institute for Liver and Intestinal Research, Amsterdam Gastroenterology & Metabolism Amsterdam University Medical Centers Amsterdam the Netherlands

2. Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases Hannover Medical School Hannover Germany

3. 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

4. Service des Maladies Congénitales et Héréditaires (Department of Hereditary and Congenital Disorders) Hôpital Charles Nicolle (Charles Nicolle Hospital) Tunis Tunisia

5. Department of Hereditary Diseases of Hepatic Metabolism Hôpital Antoine Béclère Clamart France

6. Clinic for Special Children Strasburg Pennsylvania USA

7. Department of Pediatrics Sidney Kimmel Medical College at Thomas Jefferson University Philadelphia Pennsylvania USA

8. Department of Gastroenterology and Hepatology Erasmus University Medical Center, Sophia Children's Hospital Rotterdam the Netherlands

9. Department of Pediatric Gastroenterology Erasmus University Medical Center, Sophia Children's Hospital Rotterdam the Netherlands

10. Department of Translational Medicine Federico II University of Naples Naples Italy

11. Third Military Medical University Chongqing China

12. Department of Pediatric Gastroenterology Baskent University Faculty of Medicine Ankara Turkey

13. Metabolomics and Genomics Research Center, Endocrinology and Metabolism Molecular‐Cellular Sciences Institute Tehran University of Medical Sciences Tehran Iran

14. Health research Institute, Research Center of Thalassemia & Hemoglobinopathy Ahvaz Jundishapur University of Medical Sciences Ahvaz Iran

15. Department of Pediatrics and Pediatric Hepatology, Faculty of Medicine Cairo University Cairo Egypt

16. Paediatric Gastroenterology and Nutrition Shaare Zedek Medical Center Jerusalem Israel

17. Department of Paediatric Gastroenterology Azienda Ospedaliera Papa Giovanni XXIII Bergamo Italy

18. Spark Therapeutics Philadelphia Pennsylvania USA

19. Telethon Institute of Genetics and Medicine Pozzuoli Italy

20. Departments of Pediatrics and Molecular, Cell & Cancer Biology University of Massachusetts School of Medicine Worcester Massachusetts USA

Funder

Horizon 2020 Framework Programme

ZonMw

Publisher

Wiley

Subject

Hepatology

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