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[43,44] However, the immediate-release formulations frequent dosing may be less convenient, affecting patient compliance

The gene therapy is specifically approved for those patients who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or repeated, serious spontaneous bleeding episodes and do not have neutralizing antibodies to the adeno-associated virus serotype Rh74var (AAVRh74var) capsid

Some claims may be exaggerated