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Paroxysmal nocturnal hemoglobinuria is a genetic disorder when a part of the immune system attacks red blood cells. Eculizumab is an antibody that selectively blocks the activation of the part of the immune system responsible.
Patients given eculizumab required no blood transfusions compared with the average 10 units of blood given to those on placebo. Also, hemoglobin stabilization was achieved in 49 percent of the eculizumab patients, compared with no patients in the placebo group.