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Mahlangu, Johnny; Kaczmarek, Radoslaw; von Drygalski, Annette; Shapiro, Susan; Chou, Sheng-Chieh; 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.
The New England journal of medicine, 02/2023, Letnik: 388, Številka: 8Journal Article
Participants with hemophilia A received B-domain–deleted factor VIII gene therapy delivered in an AAV5 vector. A decrease in annualized bleeding rates was maintained for 2 years despite declining factor VIII levels.
