Expanding Complement Therapeutics for the Treatment of Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH) is widely regarded as an archetypal complement-mediated disorder that has propelled complement drug discovery in recent decades. Its pathology is driven by chronic complement dysregulation resulting from the lack of the glycosyl phosphatidyl inositol-linked regulators DAF and CD59 on susceptible erythrocytes. This complement imbalance fuels persistent C3 activation on affected erythrocytes, which culminates in chronic complement-mediated intravascular hemolysis.

Source: Seminars in Hematology - February 15, 2018 Category: Hematology Authors: Dimitrios C. Mastellos, Edimara S. Reis, Despina Yancopoulou, Antonio M. Risitano, John D. Lambris Tags: Review Source Type: research