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This volume reviews the fundamental understanding of this potentially life-threatening disease and the advances in treatment that have been achieved with the use of the monoclonal antibody eculizumab.
1 A History of Research of PNH: Defining a Disease.- Part I Basic Sciences in PNH.- 2 Synthesis, genetics and congenital diseases of GPI-anchored proteins.- 3 Animal models of paroxysmal nocturnal hemoglobinuria.- 4 Complement and PNH.- Part II Clinical Sciences in PNH.- 5 Epidemiology in PNH: The PNH Global Registry.- 6 Epidemiology in PNH; Clinical Epidemiological Studies in Korea.- 7 Hemolysis in PNH; Depletion of nitric oxide.- 8 Bone Marrow Failure in PNH.- 9 Thrombophilia in PNH.- 10 Diagnosis and Classification of PNH.- 11 Clinical significance of a small population of glycosyl phosphatidylinositol-anchored membrane proteins (GPI-APs)-deficient cells in the management of bone marrow failure.- 12 CLONAL ORIGIN AND CLONAL SELECTION IN PNH.- 13 Pathogenesis of clonal dominance in PNH: Selection mechanisms in PNH.- 14 Pathogenesis of Clonal Dominance in PNH; Growth Advantage in PNH.- 15 Clinical Management in PNH.- 16 CLINICAL EFFECTS OF ECULIZUMAB IN PNH.- 17 Clinical Effects ofEculizumab in PNH; Extravascular Hemolysis after Eculizumab Treatment.- 18 Clinical Effects of Eculizumab in PNH; Poor-Responders to Eculizumab.- 19 Hematopoietic Stem Cell Transplantation in PNH.- 20 Future strategies of complement inhibition in paroxysmal nocturnal hemoglobinuria.- 21 Pregnancy in paroxysmal nocturnal hemoglobinuria.
Ajit Varki, Ajit Varki, Richard D Cummings, Jeffrey D Esko, Pamela Stanley, Gerald W Hart, Markus Aebi, Debra Mohnen, Taroh Kinoshita, Nicolle H Packer, James H Prestegard, Ronald L Schnaar, Peter H Seeberger
Ajit Varki, Richard D Cummings, Jeffrey D Esko, Pamela Stanley, Gerald Hart, Markus Aebi, Alan G Darvill, Taroh Kinoshita, Nicolle H Packer, James J Prestegard, Ronald L Schnaar, Peter H Seeberger