Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial

Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial

Background: Valoctocogene roxaparvovec, an adeno-associated virus-mediated gene therapy for severe hemophilia A, enables endogenous factor (F)VIII expression and provides bleed protection. Objectives: Determine valoctocogene roxaparvovec durability, efficacy, and safety 4 years after treatment. Meth...

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Main Authors: Andrew D. Leavitt, Johnny Mahlangu, Priyanka Raheja, Emily Symington, Doris V. Quon, Adam Giermasz, Maria Fernanda López Fernández, Gili Kenet, Gillian Lowe, Nigel S. Key, Carolyn M. Millar, Steven W. Pipe, Bella Madan, Sheng-Chieh Chou, Robert Klamroth, Jane Mason, Hervé Chambost, Flora Peyvandi, Elaine Majerus, Dominic Pepperell, Christine Rivat, Hua Yu, Tara M. Robinson, Margareth C. Ozelo
Format: Article
Language:English
Published: Elsevier 2024-11-01
Series:Research and Practice in Thrombosis and Haemostasis
Subjects:
adeno-associated virus
clinical trial
gene therapy
hemophilia A
quality of life
Online Access:http://www.sciencedirect.com/science/article/pii/S2475037924003108
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