Journal article
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1
Abstract
BACKGROUND: Severe hemophilia A (HA) negatively impacts health-related quality of life (HRQOL).
OBJECTIVES: We aimed to analyze HRQOL in adult men with severe HA without inhibitors after valoctocogene roxaparvovec gene transfer in the phase 3 trial GENEr8-1.
Authors
O'Mahony B; Dunn AL; Leavitt AD; Peyvandi F; Ozelo MC; Mahlangu J; Peerlinck K; Wang J-D; Lowe GC; Tan CW
Journal
Journal of Thrombosis and Haemostasis, Vol. 21, No. 12, pp. 3450–3462
Publisher
Elsevier
Publication Date
December 2023
DOI
10.1016/j.jtha.2023.08.032
ISSN
1538-7933