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Review Article| Volume 26, ISSUE 4, P691-708, November 2006

Novel Therapies for Hereditary Angioedema

      Despite remarkable progress in understanding the molecular basis of C1 inhibitor (C1INH) deficiency (see article by Wagenaar-Bos and Hack elsewhere in this issue) and the mechanism of swelling in hereditary angioedema (HAE) (see article by Davis elsewhere in this issue), the pharmacologic options available for the treatment of HAE in the United States have remained virtually unchanged over the past 40 years [
      • Nilsson I.M.
      • Andersson L.
      • Bjorkman S.E.
      Epsilon-aminocaproic acid (E-ACA) as a therapeutic agent based on 5 years' clinical experience.
      ,
      • Spaulding W.B.
      Methyltestosterone therapy for hereditary episodic edema (hereditary angioneurotic edema).
      ]. Prophylactic use of alpha-alkylated androgens or anti-fibrinolytic agents are generally successful in decreasing the number and severity of angioedema attacks; however, these medications are associated with significant adverse effects and do not successfully address the treatment of acute attacks. Taken together, these problems indicate a significant unmet need for improved treatment options of HAE in the United States.
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