Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long‐term data from phase III clinical trials-Reference-Cited by-同舟云学术

Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long‐term data from phase III clinical trials

Published:2015-06-22 Issue:7 Volume:90 Page:584-591
ISSN:0361-8609
Container-title:American Journal of Hematology
language:en
Short-container-title:Am. J. Hematol.

Author:

Hughes Derralynn A.¹²,Gonzalez Derlis E.³,Lukina Elena A.⁴,Mehta Atul¹²,Kabra Madhulika⁵,Elstein Deborah⁶,Kisinovsky Isaac⁷,Giraldo Pilar⁸⁹,Bavdekar Ashish¹⁰,Hangartner Thomas N.¹¹,Wang Nan¹²,Crombez Eric¹³,Zimran Ari⁶

Affiliation:

1. Department of HaematologyRoyal Free HospitalLondon United Kingdom

2. Department of HaematologyUniversity College LondonLondon United Kingdom

3. Instituto Privado de Hematología e Investigación Clínica – IPHICAsunción Paraguay

4. Department of Orphan DiseasesHematology Research CenterMoscow Russia

5. Pediatrics DepartmentAll India Institute of Medical SciencesNew Delhi India

6. Gaucher ClinicShaare Zedek Medical Center, affiliated with the Hebrew University‐Hadassah Medical SchoolJerusalem Israel

7. Hematology DepartmentYour Health SABuenos Aires Argentina

8. Medicina Metabólica HereditariaCentro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER)Zaragoza Spain

9. Grupo de Estudio de Enfermedades Hematologícas y MetabolicasHospital Universitario Miguel ServetZaragoza Spain

10. Pediatric Gastroenterology DepartmentKing Edward Memorial Hospital Research CentrePune India

11. Department of Biomedical Industrial & Human Factors EngineeringWright State UniversityDayton Ohio

12. Biostatistics & Statistical Programming DepartmentShireLexington Massachusetts

13. Rare Disease UnitShireLexington Massachusetts

Funder

Shire

Publisher

Wiley

Subject

Hematology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/ajh.24012

Reference42 articles.

1. Gaucher disease: clinical profile and therapeutic developments

2. Effect of Enzyme Replacement Therapy With Imiglucerase on BMD in Type 1 Gaucher Disease

3. Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment

4. Eight-Year Clinical Outcomes of Long-Term Enzyme Replacement Therapy for 884 Children With Gaucher Disease Type 1

Cited by 42 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry;Journal of Clinical Medicine;2024-06-19

2. A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study;Journal of Clinical Medicine;2024-05-16

3. 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

4. Assessing the diagnostic utility of the Gaucher Earlier Diagnosis Consensus (GED-C) scoring system using real-world data;Orphanet Journal of Rare Diseases;2024-02-16

5. A retrospective and prospective observational study of MRI changes in bone in patients with type 1 Gaucher disease treated with velaglucerase alfa: the EIROS study.;2024-02-12