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The goal of treatment for patients with hemophilia B (Christmas disease, factor IX deficiency) is to control bleeding and prevent joint destruction. This is accomplished by regular intravenous infusions of factor IX concentrate. Current research is focused on reducing the frequency of infusions by extending the half-life of recombinant factor IX.
To examine the safety and efficacy of a long-acting recombinant factor IX (nonacog beta pegol) prepared by site-directed glycoPEGylation, investigators conducted an industry-sponsored, multinational, randomized, single-blind, phase III clinical trial involving 67 patients with hemophilia B (factor IX, ≤2 IU/dL). Patients received weekly prophylactic doses of nonacog beta pegol (10 IU/kg or 40 IU/kg)…