Recent advances in the pathogenesis and treatment of paroxysmal nocturnal hemoglobinuria

Abstract excerpt

Paroxysmal nocturnal hemoglobinuria (PNH) is a very rare disease that has been investigated for over one century and has revealed unique aspects of the pathogenesis and pathophysiology of a hemolytic anemia. PNH results from expansion of a clone of hematopoietic cells that, as a consequence of an inactivating mutation of the X-linked gene PIG-A , are deficient in glycosylphosphatidylinositol (GPI)-linked...

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