Michael Makris: The 4-Year Results From the Hemgenix Study in Patients Without Neutralising Antibodies Published in RPTH Journal
Michael Makris, Emeritus Professor of Haemostasis and Thrombosis at the University of Sheffield, shared on LinkedIn:
“The natural history of gene therapy for hemophilia B is very different from that in hemophilia A. Following FIX gene therapy with Hemgenix, the FIX levels remain stable.
The 4-year results from the Hemgenix study in patients without neutralising antibodies have just been published in the journal Research and Practice in Thrombosis and Haemostasis.
The mean factor IX level after 4 years was 39%.
The PDF of the paper is freely available here.”
Title: Etranacogene dezaparvovec in participants with hemophilia B and without adenoassociated virus serotype 5 neutralizing antibodies: A 4-year subgroup analysis (HOPE-B)
Authors: Priyanka Raheja, Niamh O’Connell, Peter Verhamme, Peter Kampmann, Richard S. Lemons, Fei Wang, Sean Gill, Paul E. Monahan, Sandra Le Quellec, Frank W.G. Leebeek
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