Rare Disease Spotlight: Paroxysmal Nocturnal Hemoglobinuria

Rebekah Harrison

Paroxysmal nocturnal hemoglobinura (PNH) is an acquired rare disorder characterized the complement system destructing red blood cells to early. Red blood cells are missing the PIG-A gene that allows glycosyl-phosphatidylinositol (GPI) to help certain proteins stick to cells. PNH is the only hemolytic anemia caused by an acquired (rather than inherited) intrinsic defect in the cell membrane.
The worldwide prevalence is estimated in the range of 1–5 cases per million.




Medical Treatment  
Surgical Treatment


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