HEALTH-RELATED QUALITY-OF-LIFE OUTCOMES 4-YEARS AFTER TREATMENT WITH VALOCTOCOGENE ROXAPARVOVEC

Objectives: Valoctocogene roxaparvovec, a gene therapy for severe Hemophilia A (HA), helps prevent bleeding by providing the body with genetic instructions for making factor VIII (FVIII) protein. We report findings from the GENEr8-1 study 4-years after participants received valoctocogene roxaparvove...

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Bibliographic Details

Published in:	Hematology, Transfusion and Cell Therapy
Main Authors:	FP Careta, B Madan, MC Ozelo, G Kenet, S Chou, S Pipe, A Leavitt, A Ruiz, E Dashiell-Aje, J Mahlangu
Format:	Article
Language:	English
Published:	Elsevier 2024-10-01
Online Access:	http://www.sciencedirect.com/science/article/pii/S2531137924013051