Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS)-Reference-Cited by-同舟云学术

Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS)

Published:2017-12-12 Issue:2 Volume:93 Page:205-212
ISSN:0361-8609
Container-title:American Journal of Hematology
language:en
Short-container-title:Am J Hematol

Author:

Zimran Ari¹^ORCID,Belmatoug Nadia²,Bembi Bruno³,Deegan Patrick⁴,Elstein Deborah⁵,Fernandez-Sasso Diego⁶,Giraldo Pilar⁷⁸⁹¹⁰,Goker-Alpan Ozlem¹¹,Lau Heather¹²,Lukina Elena¹³,Panahloo Zoya⁵,Schwartz Ida Vanessa D.¹⁴¹⁵,

Affiliation:

1. Gaucher Clinic, Shaare Zedek Medical Center, affiliated with the Hebrew University-Hadassah Medical School; Jerusalem Israel

2. Centre de Référence des Maladies Lysosomales; Hôpitaux universitaires Paris Nord Val de Seine, Assistance Publique-Hôpitaux de Paris, Hôpital Beaujon, Service de Médecine Interne; Clichy France

3. Regional Coordinator Centre for Rare Diseases; University Hospital “Santa Maria della Misericordia”; Udine Italy

4. Department of Medicine; University of Cambridge and Lysosomal Disorders Unit; Cambridge UK

5. Shire; Zug Switzerland

6. Instituto William Osler; Buenos Aires Argentina

7. Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Instituto Salud Carlos III; Zaragoza Spain

8. Translational Research Unit, Aragon Institute of Health Research (IISAragon); Zaragoza Spain

9. Spanish Foundation for the Study and Therapy of Gaucher Disease (FEETEG); Zaragoza Spain

10. Unidad de Investigación Traslacional, Pta Baja, Hospital Universitario Miguel Servet; Zaragoza Spain

11. Lysosomal Disorders Unit and Center for Clinical Trials, O&O Alpan, LLC; Fairfax VA USA

12. New York University Langone Medical Center; New York NY USA

13. Department of Orphan Diseases; National Research Center for Hematology; Moscow Russia

14. Hospital de Clínicas de Porto Alegre; Porto Alegre Brazil

15. Department of Genetics; Universidade Federal do Rio Grande do Sul; Brazil

Funder

Shire Human Genetic Therapies Inc.

Publisher

Wiley

Subject

Hematology

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/ajh.24957/fullpdf

Reference27 articles.

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2. Gaucher disease epidemiology and natural history: a comprehensive review of the literature;Nalysnyk;Hematology.,2017

3. Replacement therapy for inherited enzyme deficiency-macrophage-targeted glucocerebrosidase for Gaucher's disease;Barton;N Engl J Med.,1991

4. Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources;Grabowski;Ann Intern Med.,1995

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