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Research and Practice in Thrombosis and Haemostasis|December 17, 2024
Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trialAndrew D Leavitt, Johnny Mahlangu, Priyanka Raheja, et al.Journal of Thrombosis and Haemostasis : JTH|April 13, 2024
Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia ABella Madan, Margareth C Ozelo, Priyanka Raheja, et al.Journal of Thrombosis and Haemostasis : JTH|September 7, 2023
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1Brian O'Mahony, Amy L Dunn, Andrew D Leavitt, et al.Journal of Thrombosis and Haemostasis : JTH|February 12, 2023
Heterogeneity in the half-life of factor VIII concentrate in patients with hemophilia A is due to variability in the clearance of endogenous von Willebrand factorEinas Elsheikh, Michelle Lavin, Lilian Antunes Heck, et al.The New England Journal of Medicine|February 22, 2023
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia AJohnny Mahlangu, Radoslaw Kaczmarek, Annette von Drygalski, et al.Haemophilia : the Official Journal of the World Federation of Hemophilia|April 27, 2022
Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitorsSteven W Pipe, Cédric Hermans, Meera Chitlur, et al.Expert Review of Hematology|March 15, 2023
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and fundingMargaret V Ragni, Guy Young, Glaivy Batsuli, et al.The New England Journal of Medicine|February 22, 2023
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia BSteven W Pipe, Frank W G Leebeek, Michael Recht, et al.Pageof 11