PNH is a rare, life-threatening hematologic disease caused by an acquired mutation in the PIG-A gene. This mutation results in red blood cells lacking GPI-anchored proteins (CD55 and CD59), which leads to complement-mediated hemolysis. C5, a key protein in the complement system, drives intravascular hemolysis (IVH), resulting in the hallmark symptoms and effects of PNH, such as1:
C5 Inhibitors as the Established Standard of Care in PNH2
Why are people with PNH unsatisfied with their current treatments?
C5 = complement protein 5; GPI = glycosylphosphatidylinositol; PIG-A = phosphatidylinositol glycan anchor biosynthesis class A; PNH = paroxysmal nocturnal hemoglobinuria.
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