EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks

Author:

Gouya Laurent123,Ventura Paolo4,Balwani Manisha5ORCID,Bissell D. Montgomery6,Rees David C.7,Stölzel Ulrich8,Phillips John D.9,Kauppinen Raili10,Langendonk Janneke G.11,Desnick Robert J.5,Deybach Jean‐Charles123,Bonkovsky Herbert L.12ORCID,Parker Charles9,Naik Hetanshi5,Badminton Michael13,Stein Penelope E.7,Minder Elisabeth14,Windyga Jerzy15,Bruha Radan16,Cappellini Maria Domenica17,Sardh Eliane18,Harper Pauline18,Sandberg Sverre19,Aarsand Aasne K.19,Andersen Janice19,Alegre Félix20,Ivanova Aneta21,Talbi Neila123,Chan Amy22,Querbes William22,Ko John22,Penz Craig22,Liu Shangbin22,Lin Tim22,Simon Amy22,Anderson Karl E.23

Affiliation:

1. Centre de Référence Maladies Rares Porphyries Colombes France

2. University of Paris Paris France

3. Laboratory of Excellence GR‐Ex Paris France

4. Università degli Studi di Modena e Reggio Emilia Emilia‐Romagna Italy

5. Icahn School of Medicine at Mount Sinai New York NY

6. University of California San Francisco CA

7. King’s College Hospital King’s College London London UK

8. Klinikum Chemnitz Porphyria Center Chemnitz Germany

9. University of Utah Salt Lake City UT

10. University Hospital of Helsinki Helsinki Finland

11. Porphyria Center Center for Lysosomal and Metabolic Disease Department of Internal Medicine Erasmus MC University Medical Center Rotterdam the Netherlands

12. Section on Gastroenterology & Hepatology Wake Forest University/NC Baptist Medical Center Winston‐Salem NC

13. University Hospital of Wales Cardiff UK

14. Stadtspital Triemli Zentrallabor Zurich Switzerland

15. Department of Hemostatic Disorders and Internal Medicine Institute of Hematology and Transfusion Medicine Warsaw Poland

16. 4th Internal Clinic General University Hospital Charles University Prague Czech Republic

17. Fondazione IRCCS, Ca Granda University of Milan Milan Italy

18. Porphyria Centre Sweden, Centre for Inherited Metabolic Diseases Karolinska Institutet, Karolinska University Hospital Stockholm Sweden

19. Norwegian Porphyria Centre Haukeland University Hospital Bergen Norway

20. Clinica Universidad de Navarra Navarra Spain

21. St. Ivan Rilski U Hospital Sofia Bulgaria

22. Alnylam Pharmaceuticals Cambridge MA

23. University of Texas Medical Branch Galveston TX

Funder

Alnylam Pharmaceuticals

Publisher

Wiley

Subject

Hepatology

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