A SIMULATION STUDY TO PROVIDE GUIDANCE FOR INDIVIDUALS TRANSITIONING FROM EMICIZUMAB TO VALOCTOCOGENE ROXAPARVOVEC

Aim: Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is a gene therapy evaluated in the phase 3 GENEr8-1 trial that provides endogenous factor VIII (FVIII) production to prevent bleeding in people with severe hemophilia A and represents an alternative to emicizumab, a recombinant antibody mimicking the...

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Bibliographic Details

Published in:	Hematology, Transfusion and Cell Therapy
Main Authors:	W Kuwabara, D Osmond, J Henshaw, S Agarwal, V Newman, C Hermans, W Miesbach, S Pipe, RS Jr, F Peyvandi
Format:	Article
Language:	English
Published:	Elsevier 2023-10-01
Online Access:	http://www.sciencedirect.com/science/article/pii/S2531137923010167