Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial-Reference-Cited by-同舟云学术

Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial

Published:2017-10-03 Issue:11 Volume:92 Page:1170-1176
ISSN:0361-8609
Container-title:American Journal of Hematology
language:en
Short-container-title:Am J Hematol

Author:

Mistry Pramod K.¹^ORCID,Lukina Elena²,Ben Turkia Hadhami³,Shankar Suma P.⁴,Baris Hagit⁵⁶⁷,Ghosn Marwan⁸,Mehta Atul⁹,Packman Seymour¹⁰,Pastores Gregory¹¹,Petakov Milan¹²,Assouline Sarit¹³,Balwani Manisha¹⁴,Danda Sumita¹⁵,Hadjiev Evgueniy¹⁶,Ortega Andres¹⁷,Gaemers Sebastiaan J. M.¹⁸,Tayag Regina¹⁹,Peterschmitt M. Judith²⁰

Affiliation:

1. Department of Internal Medicine and Pediatrics; Yale University School of Medicine; New Haven CT USA

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

3. Department of Pediatrics; Hôpital La Rabta; Tunis Tunisia

4. Department of Human Genetics; Emory University School of Medicine; Atlanta GA USA

5. Raphael Recanati Genetic Institute; Rabin Medical Center; Petach Tikvah Israel

6. Sackler Faculty of Medicine; Tel Aviv University; Tel Aviv Israel

7. The Genetics Institute, Rambam Health Care Campus and The Ruth and Bruce Rappaport Faculty of Medicine; Technion - Israel Institute of Technology; Haifa Israel

8. Department of Hematology-Oncology; Hôtel-Dieu de France University Hospital; Beirut Lebanon

9. Department of Haematology, The Royal Free Hospital; University College; London UK

10. Department of Pediatrics; UCSF School of Medicine; San Francisco CA USA

11. Department of Neurology and Pediatrics; New York University School of Medicine; New York NY USA

12. Department of Neuroendocrinology, Clinical Center of Serbia; Belgrade University Medical School; Serbia

13. Department of Medicine, Division of Hematology; Jewish General Hospital; Montreal Quebec Canada

14. Department of Genetics and Genomic Sciences; Icahn School of Medicine at Mt. Sinai Hospital; New York NY USA

15. Department of Medical Genetics; Christian Medical College; Vellore Tamil Nadu India

16. Medical University - Sofia, Faculty of Medicine, Department of Internal diseases; UMHAT “Alexandrovska”- Clinic of Hematology; Sofia Bulgaria

17. OCA Hospital; Monterrey Mexico

18. Global Pharmacovigilance; Sanofi Genzyme; Naarden The Netherlands

19. Prometrika; Cambridge MA USA

20. Rare Diseases Clinical Development; Sanofi Genzyme; Cambridge MA USA

Funder

Sanofi Genzyme

Publisher

Wiley

Subject

Hematology

Reference17 articles.

1. Complement drives glucosylceramide accumulation and tissue inflammation in Gaucher disease;Pandey;Nature.,2017

2. Glucocerebrosidase gene-deficient mouse recapitulates Gaucher disease displaying cellular and molecular dysregulation beyond the macrophage;Mistry;Proc Natl Acad Sci USA.,2010

3. Eliglustat tartrate: glucosylceramide synthase inhibitor treatment;Shayman;Drugs Future.,2010

4. 2014

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