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Health-Related Quality of Life Following Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in the Phase 3 Trial GENEr8-1

Brian O'Mahony,Johnny Mahlangu,Kathelijne Peerlinck,Jiaan-Der Wang,Gillian Lowe,Chee Wee Tan,Adam Giermasz,Huyen A. Tran,Teh-Liane Khoo,Erin Cockrell,Dominic Pepperell,Herve Chambost,Maria Fernanda Lopez Fernandez,Rashid Kazmi,Elaine Majerus,Mark W. Skinner,Robert Klamroth,Jennifer Quinn,Hua Yu,Wing Yen Wong,Adebayo Lawal,Tara M. Robinson,Benjamin Kim

Journal of thrombosis and haemostasis（2023）

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关键词
gene therapy,health-related quality of life,hemophilia A,patient-reported outcome,valoctocogene roxaparvovec
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