Paroxysmal nocturnal hemoglobinuria, caused by somatic mutations in the X-linked phosphatidylinositol glycan class A (PIGA) gene involved in glycosylphosphatidylinositol (GPI) anchor biosynthesis (3), is characterized by failure of membrane anchorage of GPI-anchored proteins, including complement regulatory proteins, determining complement-mediated cell lysis that remains, to date, the only pharmacologically targeted pathway for the treatment of PNH (1, 5, 33). This evidence concerns the gene PIGA and paroxysmal nocturnal hemoglobinuria.