About this Medicine article
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A by Johnny Mahlangu; Radoslaw Kaczmarek; Annette von Drygalski; Susan Shapiro; Sheng-Chieh Chou; Margareth C. Ozelo; Gili Kenet; Flora Peyvandi; Michael Wang; Bella Madan; Nigel S. Key; Michael Laffan; Amy L. Dunn; Jane Mason; Doris V. Quon; Emily Symington; Andrew D. Leavitt; Johannes Oldenburg; Hervé Chambost; Mark T. Reding; Kala Jayaram; Hua Yu; Reena Mahajan; Konstantia-Maria Chavele; Divya B. Reddy; Joshua Henshaw; Tara M. Robinson; Wing Yen Wong; Steven W. Pipe is a Medicine article available to read on EtoBox.
BACKGROUND Valoctocogene roxaparvovec delivers a B-domain-deleted factor VIII coding sequence with an adeno-associated virus vector to prevent bleeding in persons with severe hemophilia A. The findings of a phase 3 study of the efficacy and safety of valoctocogene roxaparvovec therapy evaluated after 52 weeks in men with severe hemophilia A have been published previously.
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- Author
- Johnny Mahlangu; Radoslaw Kaczmarek; Annette von Drygalski; Susan Shapiro; Sheng-Chieh Chou; Margareth C. Ozelo; Gili Kenet; Flora Peyvandi; Michael Wang; Bella Madan; Nigel S. Key; Michael Laffan; Amy L. Dunn; Jane Mason; Doris V. Quon; Emily Symington; Andrew D. Leavitt; Johannes Oldenburg; Hervé Chambost; Mark T. Reding; Kala Jayaram; Hua Yu; Reena Mahajan; Konstantia-Maria Chavele; Divya B. Reddy; Joshua Henshaw; Tara M. Robinson; Wing Yen Wong; Steven W. Pipe
- Publisher
- Massachusetts Medical Society
- Published
- 2023
- Language
- EN
- Field
- Medicine (Health Sciences)