Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A-Reference-Cited by-同舟云学术

Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A

Published:2024-07 Issue:7 Volume:22 Page:1880-1893
ISSN:1538-7836
Container-title:Journal of Thrombosis and Haemostasis
language:en
Short-container-title:Journal of Thrombosis and Haemostasis

Author:

Madan Bella,Ozelo Margareth C.,Raheja Priyanka,Symington Emily,Quon Doris V.,Leavitt Andrew D.,Pipe Steven W.,Lowe Gillian,Kenet Gili,Reding Mark T.,Mason Jane,Wang Michael,von Drygalski Annette,Klamroth Robert,Shapiro Susan,Chambost Hervé,Dunn Amy L.,Oldenburg Johannes,Chou Sheng-Chieh,Peyvandi Flora,Millar Carolyn M.,Osmond Dane,Yu Hua,Dashiell-Aje Ebony,Robinson Tara M.,Mahlangu Johnny

Funder

BioMarin Pharmaceutical Inc

Publisher

Elsevier BV

Reference46 articles.

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4. Haemophilia Experiences, Results and Opportunities (HERO) study: treatment-related characteristics of the population;Nugent;Haemophilia,2015

5. The impact of severe haemophilia and the presence of target joints on health-related quality-of-life;O'Hara;Health Qual Life Outcomes,2018

Cited by 5 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. Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A;Blood Advances;2024-08-30

2. Normalisation of Haemostasis in Haemophilia A;EMJ Hematology;2024-07-25

3. A Debate on the Use of Gene Therapy in Patients with Haemophilia;EMJ Hematology;2024-07-25

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