References in periodicals archive ?
The Orphan Drug Designation is granted for the treatment and prophylaxis of bleeding episodes in patients with congenital factor IX deficiency (hemophilia B).
This total solution would be particularly useful for research purposes and could potentially be used to treat protein deficiency diseases such as anemia, which results from erythropoietin (EPO) deficiency, and hemophilia, which results from factor IX deficiency.
Factor IX deficiency is an expensive disease to treat.