Single-dose gene therapy is potentially life-changing for adults with hemophilia B

Single-dose gene therapy is potentially life-changing for adults with hemophilia B

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71% after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published in the New England Journal of Medicine by researchers from the University of Pennsylvania Perelman School of Medicine and a multicenter group of investigators.

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