Author(s): Anita Hill, Hubert Schrezenmeier, Peter Hillmen, et al. Abstract: PF305 Session topic: 12. Bone marrow failure syndromes incl. PNH – Clinical Background Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, clonal, hematopoietic stem cell disorder caused by a deficiency of glycosylphosphatidylinositol-anchored proteins (GPI-AP) on cell surfaces. Patients with acquired mutations in the phosphatidylinositol glycan...
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