Another Victory for Patients with Hemophilia

Congenital hemophilia A is a rare bleeding disorder caused by a mutation in the gene encoding factor VIII, resulting in a deficiency of factor VIII activity. Severe hemophilia (factor VIII activity level, <1%) is characterized by repetitive bleeding into joints beginning in early childhood and poses a major risk of life-threatening hemorrhage. Moderate hemophilia (factor VIII activity level, 1 to 5%) is associated with less frequent joint and soft-tissue bleeding related to mild trauma. Replacement therapy with the use of factor VIII concentrates restores hemostasis by raising levels of factor VIII activity and is effective in the treatment of . . .