Three-year Outcomes of Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

Bella Madan,Margareth C. Ozelo,Priyanka Raheja,Emily Symington,Doris V. Quon,Andrew D. Leavitt,Steven W. Pipe,Gillian Lowe,Gili Kenet,Mark T. Reding,Jane Mason,Michael Wang,Annette von Drygalski,Robert Klamroth,Susan Shapiro,Herve Chambost,Amy L. Dunn,Johannes Oldenburg,Sheng-Chieh Chou,Flora Peyvandi,Carolyn M. Millar,Dane Osmond,Hua Yu, Ebony Dashiell-Aje,Tara M. Robinson,Johnny Mahlangu

JOURNAL OF THROMBOSIS AND HAEMOSTASIS（2024）

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关键词
adeno-associated virus,clinical trial,gene therapy,health-related quality of life,hemophilia A,phase 3
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