https://doi.org/10.4081/btvb.2022.32
Gene transfer in hemophilia A: not cogent yet
Published: 20 April 2022
Abstract Views: 1965
PDF: 209
Publisher's note
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.
Authors
The phase 3 clinical study of gene transfer in hemophilia A using the BioMarin vector AAV5-hFVIII-SQ (also identified as valoctocogene roxaparvovec) recently reported the results obtained in as many as 134 adult men with severe hemophilia A, so that it is at the moment the largest gene transfer study ever conducted in a rare monogenic disorder [...].
Ozelo MC, Mahlangu J, Pasi KJ, et al. Valoctocogene roxaparvovec gene therapy for hemophilia A. N Engl J Med 2022;386:1013-25.
DOI: https://doi.org/10.1056/NEJMoa2113708
Thornburg CD. Prepare the way for hemophilia A gene therapy. N Engl J Med 2022;386:1081-2.
DOI: https://doi.org/10.1056/NEJMe2200878
Mannucci PM. Miracle of haemophilia drugs: personal views about a few main players. Haemophilia 2018;24:557-52.
DOI: https://doi.org/10.1111/hae.13519
Mannucci PM. Treatment of hemophilia - more amazing progress. N Engl J Med 2020;383:1068-70.
DOI: https://doi.org/10.1056/NEJMe2024545
Konkle BA, Shapiro AD, Quon DV, et al. BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A. N Engl J Med 2020;383:1018-27.
DOI: https://doi.org/10.1056/NEJMoa2002699
Peyvandi F, Garagiola I, Mannucci PM. Post-authorization pharmacovigilance for hemophilia in Europe and the USA: independence and transparency are keys. Blood Rev 2021;49:100828.
DOI: https://doi.org/10.1016/j.blre.2021.100828
How to Cite
Mannucci, P. M. (2022). Gene transfer in hemophilia A: not cogent yet. Bleeding, Thrombosis and Vascular Biology, 1(1). https://doi.org/10.4081/btvb.2022.32
Copyright (c) 2022 The Author(s)
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License.
PAGEPress has chosen to apply the Creative Commons Attribution NonCommercial 4.0 International License (CC BY-NC 4.0) to all manuscripts to be published.
Most read articles by the same author(s)
- Pier Mannuccio Mannucci, An ecological alliance against air pollution and cardiovascular disease , Bleeding, Thrombosis and Vascular Biology: Vol. 1 No. 1 (2022)
- Pier Mannuccio Mannucci, Growing weapons to fight hemophilia , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 1 (2023)
Similar Articles
- Anne Eichmann, The Lefoulon Delalande Foundation honors the lymphatic vascular system , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 4 (2023)
- Roberto Mario Santi, Annamaria Fulghesu, Ezio Zanon, Erica De Candia, Elvira Grandone, Giancarlo Di Renzo, Claudia Succu, Valentina Tosto, Vincenzina Bruni, Paolo Gresele, Diagnosis and management of abnormal uterine bleeding in adolescence , Bleeding, Thrombosis and Vascular Biology: Vol. 3 No. 1 (2024)
- Daniela Poli, Walter Ageno, Emilia Antonucci, Salvatore Bradamante, Eugenio Bucherini, Paolo Chiarugi, Antonio Chistolini, Benilde Cosmi, Anna Falanga, Antonio Insana, Domenico Lione, Rosa Maria Lombardi, Giuseppe Malcangi, Rossella Marcucci, Giuliana Martini, Lucilla Masciocco, Carmelo Paparo, Daniele Pastori, Simona Pedrini, Vittorio Pengo, Pasquale Pignatelli, Andrea Toma, Sophie Testa, Gualtiero Palareti, Management of anticoagulation in atrial fibrillation patients in Italy: insight from the Atrial Fibrillation-Survey on Anticoagulated Patients Register (AF-START) , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 2 (2023)
- Valerio De Stefano, Science and scientists under attack , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 2 (2023)
- Chiara Tonelli, A contribution from Umberto Veronesi Foundation , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 1 (2023)
- Gaetano Torlone, The metaphor of Ukrainian science “bleeding” , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 1 (2023)
- Daniela Poli, Riccardo Tartaglia, Doris Barcellona , Paolo Bucciarelli , Antonio Ciampa, Elvira Grandone, Giuseppe Malcangi, Giuseppe Rescigno, Vincenzo Toschi, Sophie Testa, Alessandro Squizzato, Attitude to clinical research among health professionals affiliated with the Italian Federation of Centers for the Diagnosis of Thrombotic Disorders and the Surveillance of the Antithrombotic Therapies (FCSA) , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 1 (2023)
- Boris Shenkman, Ivan Budnik, Yulia Einav, Interaction between adenosine diphosphate receptors and protein-kinase C isoforms in platelet adhesion under flow condition , Bleeding, Thrombosis and Vascular Biology: Vol. 2 No. 1 (2023)
- Sergio Coccheri, A re-appraisal of thrombogenesis in COVID-19, seen as a multiple "Complex system" , Bleeding, Thrombosis and Vascular Biology: Vol. 1 No. 3 (2022)
- Augusto Di Castelnuovo, Licia Iacoviello, Moving beyond p-value , Bleeding, Thrombosis and Vascular Biology: Vol. 1 No. 1 (2022)
You may also start an advanced similarity search for this article.
