Early results from trials of investigational hemophilia A and B gene therapies were recently published in the New England Journal of Medicine. Both ongoing trials demonstrated substantial and sustained increases in endogenous clotting factor activity and reductions in bleeding and exogenous factor use. Hemophilia A and B are caused by genetic mutations that result in blood clotting factor VIII and IX deficiency, respectively. Hemophilia A is 6 times more common than hemophilia B.
Abbasi J. Hemophilia Gene Therapies Show Promise. JAMA. 2018;319(6):539. doi:10.1001/jama.2018.0524
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