Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

Margareth C. Ozelo; Johnny Mahlangu; K. John Pasi; Adam Giermasz; Andrew D. Leavitt; Michael Laffan; Emily Symington; Doris V. Quon; Jiaan-Der Wang; Kathelijne Peerlinck; Steven W. Pipe; Bella Madan; Nigel S. Key; Glenn F. Pierce; Brian O’Mahony; Radoslaw Kaczmarek; Joshua Henshaw; Adebayo Lawal; Kala Jayaram; Mei Huang; Xinqun Yang; Wing Y. Wong; Benjamin Kim

Journal ArticleOPEN ACCESS

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

Ozelo M
Mahlangu J
Pasi K
et al.

New England Journal of Medicine (2022) 386(11) 1013-1025

DOI: 10.1056/nejmoa2113708

343Citations

335Readers

Abstract

Abstract Background Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a li...

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APA

Ozelo, M. C., Mahlangu, J., Pasi, K. J., Giermasz, A., Leavitt, A. D., Laffan, M., … Kim, B. (2022). Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A. New England Journal of Medicine, 386(11), 1013–1025. https://doi.org/10.1056/nejmoa2113708

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

Abstract

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