Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial-Reference-Cited by-同舟云学术

Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial

Published:2018-10-26 Issue:1 Volume:94 Page:29-38
ISSN:0361-8609
Container-title:American Journal of Hematology
language:en
Short-container-title:Am J Hematol

Author:

Lukina Elena¹^ORCID,Watman Nora²,Dragosky Marta³,Lau Heather⁴,Avila Arreguin Elsa⁵,Rosenbaum Hanna⁶,Zimran Ari⁷^ORCID,Foster Meredith C.⁸,Gaemers Sebastiaan J. M.⁹,Peterschmitt M. Judith¹⁰

Affiliation:

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

2. Division of Hematology; Hospital Ramos Mejia; Buenos Aires Argentina

3. Department of Hematology; IMAI-Research; Buenos Aires Argentina

4. Department of Neurology; New York University School of Medicine; New York New York

5. Department of Hematology; Instituto Mexicano del Seguro Social Hospital de Especialidades; Col. La Raza Mexico

6. Hematology Department, Rambam Medical Center; Haifa Israel

7. Gaucher Clinic, Department of Medicine, Shaare Zedek Medical Center; Jerusalem Israel

8. Global Medical Communications; Sanofi Genzyme; Cambridge Massachusetts

9. Global Pharmacovigilance; Sanofi Genzyme; Naarden The Netherlands

10. Rare Diseases Clinical Development; Sanofi Genzyme; Cambridge Massachusetts

Funder

Sanofi Genzyme

Publisher

Wiley

Subject

Hematology

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/ajh.25300/fullpdf

Reference30 articles.

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2. A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: evaluation of frequency, timing, and duration;Peterschmitt;Blood Cells Mol Dis,2018

3. The cellular pathology of lysosomal diseases;Cox;J Pathol,2012

4. Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial;Mistry;JAMA,2015

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