Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A-Reference-Cited by-同舟云学术

Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A

Published:2021-08-11 Issue:6 Volume:27 Page:947-956
ISSN:1351-8216
Container-title:Haemophilia
language:en
Short-container-title:Haemophilia

Author:

Pasi K John¹,Laffan Michael²,Rangarajan Savita³,Robinson Tara M⁴,Mitchell Nina⁴,Lester Will⁵,Symington Emily⁶,Madan Bella⁷,Yang Xinqun⁴,Kim Benjamin⁴,Pierce Glenn F⁸,Wong Wing Yen⁴

Affiliation:

1. Barts and the London School of Medicine and Dentistry London UK

2. Centre for Haematology, Imperial College London London UK

3. University Hospital Southampton Southampton UK

4. BioMarin Pharmaceutical Inc. Novato California USA

5. University Hospitals Birmingham NHS Foundation Trust Birmingham UK

6. Cambridge University Hospitals NHS Foundation Trust Cambridge UK

7. Guy's and St Thomas’ NHS Foundation Trust London UK

8. Consultant La Jolla California USA

Publisher

Wiley

Subject

Genetics (clinical),Hematology,General Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/hae.14391

Reference26 articles.

1. WFH Guidelines for the Management of Hemophilia, 3rd edition

2. Guidelines for the management of hemophilia

3. Men with severe hemophilia in the United States: birth cohort analysis of a large national database

4. Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non‐interventional study in a real‐world setting

5. AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

Cited by 73 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. The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art;Bleeding, Thrombosis and Vascular Biology;2024-09-04

2. The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice;Molecular Therapy - Methods & Clinical Development;2024-09

3. Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A;Blood Advances;2024-08-30

4. Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A;Haemophilia;2024-07-08

5. Clinical immunogenicity outcomes from GENEr8-1, a phase 3 study of valoctocogene roxaparvovec, an AAV5-vectored gene therapy for hemophilia A;Molecular Therapy;2024-07