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

Sem J. Aronson; Norman Junge; CureCN Consortium; Mediha Trabelsi; Wided Kelmemi; Aurelie Hubert; Karlla W. Brigatti; Michael D. Fox; Robert J. de Knegt; Johanna C. Escher; Virginia M. Ginocchio; Raffaele Iorio; Yan Zhu; Figen Özçay; Fakher Rahim; Mortada H.F. El-Shabrawi; Eyal Shteyer; Angelo Di Giorgio; Lorenzo D'Antiga; Federico Mingozzi; Nicola Brunetti-Pierri; Kevin A. Strauss; Philippe Labrune; Ridha Mrad; Ulrich Baumann; Ulrich Beuers; Piter J. Bosma

doi:10.1111/liv.15239

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

Sem J. Aronson
, Norman Junge^*
, CureCN Consortium
, Mediha Trabelsi
, Wided Kelmemi
, Aurelie Hubert
, Karlla W. Brigatti
, Michael D. Fox
, Robert J. de Knegt
, Johanna C. Escher
, Virginia M. Ginocchio
, Raffaele Iorio
, Yan Zhu
, Figen Özçay
, Fakher Rahim
, Mortada H.F. El-Shabrawi
, Eyal Shteyer
, Angelo Di Giorgio
, Lorenzo D'Antiga
, Federico Mingozzi

Nicola Brunetti-Pierri, Kevin A. Strauss, Philippe Labrune, Ridha Mrad, Ulrich Baumann, Ulrich Beuers, Piter J. Bosma

^*Corresponding author for this work

Hannover Medical School
Université de Tunis El Manar
Hôpital Antoine Béclère
Clinic for Special Children
Thomas Jefferson University
University of Naples Federico II
Army Medical University
Baskent University
Tehran University of Medical Sciences
Ahvaz Jundishapur University of Medical Sciences
Cairo University
Shaare Zedek Medical Center
Azienda Ospedaliera Papa Giovanni XXIII
Spark Therapeutics
Telethon Institute of Genetics and Medicine
University of Massachusetts Medical School
Amsterdam UMC

Research output: Contribution to journal › Article › Academic › peer-review

26 Citations (Scopus)

182 Downloads (Pure)

Original language	English
Pages (from-to)	1593-1604
Number of pages	12
Journal	Liver International
Volume	42
Issue number	7
Early online date	11 Mar 2022
DOIs	https://doi.org/10.1111/liv.15239
Publication status	Published - Jul 2022

Bibliographical note

Funding Information:
This project is part of the CureCN project and has received funding from the European Union's Horizon 2020 research and innovation program under Grant Agreement No. 755225 (CureCN) and ZonMw TGO under Grant Agreement No. 43300003 (to P.J.B.).

Funding Information:
The authors would like to thank the Dutch Najjar Foundation, the French Crigler-Najjar Patient Association and the Italian Crigler-Najjar Patient Association CIAMI for their support, all physicians that collaborated to this project and included clinical data (additional to the authors: Dr. Belbouab, Mustapha Pacha University Hospital, Sidi M'Hamed, Algeria; Prof. Indolfi, Meyer Children's Hospital Florance, Italy; Prof. Pawlowska, Children's Memorial Health Institute, Warsaw, Poland) and the patients who kindly accepted to contribute to the CN World Registry. Interested to participate in the CN World Registry project? Please visit the site of one of the involved patient organizations via the CureCN homepage (https://curecn.eu/partners/cn-pa) for more information or send an email to Piter Bosma ([email protected]).

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Disease burden and management of Crigler-Najjar syndrome Report of a world registryFinal published version, 718 KBLicence: CC BY

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Aronson, S. J., Junge, N., CureCN Consortium, Trabelsi, M., Kelmemi, W., Hubert, A., Brigatti, K. W., Fox, M. D., de Knegt, R. J., Escher, J. C., Ginocchio, V. M., Iorio, R., Zhu, Y., Özçay, F., Rahim, F., El-Shabrawi, M. H. F., Shteyer, E., Di Giorgio, A., D'Antiga, L., ... Bosma, P. J. (2022). Disease burden and management of Crigler-Najjar syndrome: Report of a world registry. Liver International, 42(7), 1593-1604. https://doi.org/10.1111/liv.15239

@article{9794d118960d4e829fb0712d3edbd59d,

title = "Disease burden and management of Crigler-Najjar syndrome: Report of a world registry",

author = "Aronson, \{Sem J.\} and Norman Junge and \{CureCN Consortium\} and Mediha Trabelsi and Wided Kelmemi and Aurelie Hubert and Brigatti, \{Karlla W.\} and Fox, \{Michael D.\} and \{de Knegt\}, \{Robert J.\} and Escher, \{Johanna C.\} and Ginocchio, \{Virginia M.\} and Raffaele Iorio and Yan Zhu and Figen {\"O}z{\c c}ay and Fakher Rahim and El-Shabrawi, \{Mortada H.F.\} and Eyal Shteyer and \{Di Giorgio\}, Angelo and Lorenzo D'Antiga and Federico Mingozzi and Nicola Brunetti-Pierri and Strauss, \{Kevin A.\} and Philippe Labrune and Ridha Mrad and Ulrich Baumann and Ulrich Beuers and Bosma, \{Piter J.\}",

note = "Funding Information: This project is part of the CureCN project and has received funding from the European Union's Horizon 2020 research and innovation program under Grant Agreement No. 755225 (CureCN) and ZonMw TGO under Grant Agreement No. 43300003 (to P.J.B.). Funding Information: The authors would like to thank the Dutch Najjar Foundation, the French Crigler-Najjar Patient Association and the Italian Crigler-Najjar Patient Association CIAMI for their support, all physicians that collaborated to this project and included clinical data (additional to the authors: Dr. Belbouab, Mustapha Pacha University Hospital, Sidi M'Hamed, Algeria; Prof. Indolfi, Meyer Children's Hospital Florance, Italy; Prof. Pawlowska, Children's Memorial Health Institute, Warsaw, Poland) and the patients who kindly accepted to contribute to the CN World Registry. Interested to participate in the CN World Registry project? Please visit the site of one of the involved patient organizations via the CureCN homepage (https://curecn.eu/partners/cn-pa) for more information or send an email to Piter Bosma ([email protected]).",

year = "2022",

month = jul,

doi = "10.1111/liv.15239",

language = "English",

volume = "42",

pages = "1593--1604",

number = "7",

}

Aronson, SJ, Junge, N, CureCN Consortium, Trabelsi, M, Kelmemi, W, Hubert, A, Brigatti, KW, Fox, MD, de Knegt, RJ , Escher, JC, Ginocchio, VM, Iorio, R, Zhu, Y, Özçay, F, Rahim, F, El-Shabrawi, MHF, Shteyer, E, Di Giorgio, A, D'Antiga, L, Mingozzi, F, Brunetti-Pierri, N, Strauss, KA, Labrune, P, Mrad, R, Baumann, U, Beuers, U & Bosma, PJ 2022, 'Disease burden and management of Crigler-Najjar syndrome: Report of a world registry', Liver International, vol. 42, no. 7, pp. 1593-1604. https://doi.org/10.1111/liv.15239

TY - JOUR

T1 - Disease burden and management of Crigler-Najjar syndrome

T2 - Report of a world registry

AU - Aronson, Sem J.

AU - Junge, Norman

AU - CureCN Consortium

AU - Trabelsi, Mediha

AU - Kelmemi, Wided

AU - Hubert, Aurelie

AU - Brigatti, Karlla W.

AU - Fox, Michael D.

AU - de Knegt, Robert J.

AU - Escher, Johanna C.

AU - Ginocchio, Virginia M.

AU - Iorio, Raffaele

AU - Zhu, Yan

AU - Özçay, Figen

AU - Rahim, Fakher

AU - El-Shabrawi, Mortada H.F.

AU - Shteyer, Eyal

AU - Di Giorgio, Angelo

AU - D'Antiga, Lorenzo

AU - Mingozzi, Federico

AU - Brunetti-Pierri, Nicola

AU - Strauss, Kevin A.

AU - Labrune, Philippe

AU - Mrad, Ridha

AU - Baumann, Ulrich

AU - Beuers, Ulrich

AU - Bosma, Piter J.

N1 - Funding Information: This project is part of the CureCN project and has received funding from the European Union's Horizon 2020 research and innovation program under Grant Agreement No. 755225 (CureCN) and ZonMw TGO under Grant Agreement No. 43300003 (to P.J.B.). Funding Information: The authors would like to thank the Dutch Najjar Foundation, the French Crigler-Najjar Patient Association and the Italian Crigler-Najjar Patient Association CIAMI for their support, all physicians that collaborated to this project and included clinical data (additional to the authors: Dr. Belbouab, Mustapha Pacha University Hospital, Sidi M'Hamed, Algeria; Prof. Indolfi, Meyer Children's Hospital Florance, Italy; Prof. Pawlowska, Children's Memorial Health Institute, Warsaw, Poland) and the patients who kindly accepted to contribute to the CN World Registry. Interested to participate in the CN World Registry project? Please visit the site of one of the involved patient organizations via the CureCN homepage (https://curecn.eu/partners/cn-pa) for more information or send an email to Piter Bosma ([email protected]).

PY - 2022/7

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UR - https://www.scopus.com/pages/publications/85133100517

U2 - 10.1111/liv.15239

DO - 10.1111/liv.15239

M3 - Article

C2 - 35274801

AN - SCOPUS:85133100517

SN - 1478-3223

VL - 42

SP - 1593

EP - 1604

JO - Liver International

JF - Liver International

IS - 7

ER -

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

Bibliographical note

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