Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase-Reference-Cited by-同舟云学术

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Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase

Published:2013-01-22 Issue:3 Volume:88 Page:172-178
ISSN:0361-8609
Container-title:American Journal of Hematology
language:en
Short-container-title:Am. J. Hematol.

Author:

Zimran Ari¹,Pastores Gregory M.²,Tylki-Szymanska Anna³,Hughes Derralynn A.⁴,Elstein Deborah¹,Mardach Rebecca⁵,Eng Christine⁶,Smith Laurie⁷,Heisel-Kurth Margaret⁸,Charrow Joel⁹,Harmatz Paul¹⁰,Fernhoff Paul¹¹,Rhead William¹²,Longo Nicola¹³,Giraldo Pilar¹⁴,Ruiz Juan A.¹⁵,Zahrieh David¹⁵,Crombez Eric¹⁵,Grabowski Gregory A.¹⁶

Affiliation:

1. Shaare Zedek Medical Center and Hebrew University-Hadassah Medical School; Jerusalem; Israel

2. New York University School of Medicine; New York; New York

3. Children's Memorial Health Institute; Warsaw; Poland

4. Royal Free Hospital, University College London School of Medicine; London; United Kingdom

5. Kaiser Permanente; Los Angeles; California

6. Baylor College of Medicine; Houston; Texas

7. Children's Mercy Hospital; Kansas City; Missouri

8. Children's Hospitals of Minnesota; Minneapolis; Minnesota

9. Ann and Robert H. Lurie Children's Hospital of Chicago; Chicago; Illinois

10. Children's Hospital Oakland; Oakland; California

11. Emory University; Decatur; Georgia

12. Children's Hospital of Wisconsin; Milwaukee; Wisconsin

13. University of Utah; Salt Lake City; Utah

14. Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Hospital Universitario Miguel Servet; Zaragoza; Spain

15. Shire Human Genetic Therapies; Lexington; Massachusetts

16. Cincinnati Children's Hospital Medical Center; Cincinnati; Ohio

Publisher

Wiley

Subject

Hematology

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/ajh.23383/fullpdf

Reference23 articles.

1. Grabowski GA Petsko GA Kolodny EH The Online Metabolic and Molecular Bases of Inherited Diseases http://dx.doi.org/10.1036/ommbid.176

2. Nucleotide sequence of cDNA containing the complete coding sequence for human lysosomal glucocerebrosidase;Tsuji;J Biol Chem,1986

3. A pharmacokinetic analysis of a novel enzyme replacement therapy with Gene-Activated human glucocerebrosidase (GA-GCB) in patients with type 1 Gaucher disease;Zimran;Blood Cells Mol Dis,2007

4. Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience;Zimran;Blood,2010

5. Development of a panel of highly sensitive, equivalent assays for detection of antibody responses to velaglucerase alfa or imiglucerase enzyme replacement therapy in patients with Gaucher disease;Séllos-Moura;J Immunol Methods,2011

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2. Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry;Journal of Clinical Medicine;2024-05-09

3. Velaglucerase alfa for treatment in children with Gaucher disease type 1: the Russian experience;Pediatric Hematology/Oncology and Immunopathology;2023-10-01

4. Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders;Advanced Drug Delivery Reviews;2023-06

5. Osteonecrosis in Gaucher disease in the era of multiple therapies: Biomarker set for risk stratification from a tertiary referral center;eLife;2023-05-30

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