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An Introduction to Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) was first described as a distinct and life-threatening entity in 1882. The clinical hallmark of PNH, nocturnal hemoglobinuria, has fascinated generations of physicians and led to the discovery of the alternative complement pathway, identification of the membrane proteins that regulate complement and attachment mechanisms for cell surface proteins, and the genetic basis of the disease (Parker, 2008). PNH has traditionally been associated with significant morbidity and poor prognosis. However, approval of the anti-complement protein 5 (C5) antibody eculizumab for treatment in 2007 has significantly changed the natural history of PNH.