HEALTH-RELATED QUALITY-OF-LIFE OUTCOMES 4-YEARS AFTER TREATMENT WITH VALOCTOCOGENE ROXAPARVOVEC
FP Caretaa, B Madanb, MC Ozeloc, G Kenetd, S Choue, S Pipef, A Leavittg, A Ruizh, E Dashiell-Ajei, J Mahlanguj
a Universidade Federal do Espírito Santo (UFES), Vitória, ES, Brazil
b Guy's and St Thomas'NHS Foundation Trust, London, UK
c Centro de Hematologia e Hemoterapia (Hemocentro), Department of Internal Medicine, Faculdade de Ciências Médicas (FCM), Universidade Estadual de Campinas (UNICAMP), Campinas, SP, Brazil
d The National Hemophilia Center and Amalia Biron Research Institute of Thrombosis and Hemostasis, Sheba Medical Center, Tel Hashomer, Tel Aviv University
e Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan
f Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI, USA
g Adult Hemophilia Treatment Center, University of California San Francisco, San Francisco, CA, USA
h National Bleeding Disorders Foundation, Oakland Park, FL, USA
i BioMarin Pharmaceutical Inc., Novato, CA, USA
j Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa
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