Upgrading the evidence for the use of ambroxol in Gaucher disease and GBA related Parkinson: Investigator initiated registry based on real life data

Author:

Istaiti Majdolen1,Revel‐Vilk Shoshana12ORCID,Becker‐Cohen Michal1,Dinur Tama1ORCID,Ramaswami Uma3,Castillo‐Garcia Daniela4ORCID,Ceron‐Rodriguez Magdalena4,Chan Alicia5,Rodic Predrag67,Tincheva Radka Stefanova8,Al‐Hertani Walla9,Lee Beom Hee10,Yang Chia‐Feng1112,Kiec‐Wilk Beata1314,Fiumara Agata15,Rubio Barbara16,Zimran Ari12ORCID

Affiliation:

1. Gaucher Unit Shaare Zedek Medical Center Jerusalem Israel

2. Faculty of Medicine Hebrew University of Jeursalem Jerusalem Israel

3. Lysosomal Disorders Unit Royal Free London NHS Foundation Trust London UK

4. Department of Lysosomal Diseases Hospital Infantil de México Federico Gómez Ciudad de México Mexico

5. Department of Medical Genetics University of Alberta Edmonton Edmonton Alberta Canada

6. Department of Hematology and Oncology University Children's Hospital Belgrade Serbia

7. Faculty of Medicine University of Belgrade Beograd Serbia

8. Department of Clinical Genetics University Pediatric Hospital Sofia Bulgaria

9. Division of Genetics and Genomics Boston Children's Hospital, Harvard Medical School Boston Massachusetts

10. Department of Pediatrics, Medical Genetics Center Asan Medical Center Children's Hospital, University of Ulsan College of Medicine Seoul South Korea

11. Department of Pediatrics Taipei Veterans General Hospital Taipei Taiwan

12. School of Medicine National Yang‐Ming University Taipei Taiwan

13. Clinical Department of Metabolic Diseases and Diabetology University Hospital in Krakow Krakow Poland

14. Department of Metabolic Diseases Jagiellonian University Medical College Krakow Poland

15. Regional Referral Centre for Inborn Errors Metabolism, Paediatric Clinic, Department of Clinical and Experimental Medicine University of Catania Catania Italy

16. Paediatric Department Hospital Universitario de Getafe Madrid Spain

Funder

University of Cambridge

Publisher

Wiley

Subject

Hematology

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