Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A
Journal
The New England journal of medicine
Journal Volume
388
Journal Issue
8
Pages
694
Date Issued
2023-02-23
Author(s)
Mahlangu, Johnny
Kaczmarek, Radoslaw
von Drygalski, Annette
Shapiro, Susan
Ozelo, Margareth C
Kenet, Gili
Peyvandi, Flora
Wang, Michael
Madan, Bella
Key, Nigel S
Laffan, Michael
Dunn, Amy L
Mason, Jane
Quon, Doris V
Symington, Emily
Leavitt, Andrew D
Oldenburg, Johannes
Chambost, Hervé
Reding, Mark T
Jayaram, Kala
Yu, Hua
Mahajan, Reena
Chavele, Konstantia-Maria
Reddy, Divya B
Henshaw, Joshua
Robinson, Tara M
Wong, Wing Yen
Pipe, Steven W
Abstract
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.
Subjects
Coagulation | Genetics | Genetics General | Hematology/Oncology
SDGs
Type
journal article