Navigating the Complement Pathway to Optimize PNH Treatment with Pegcetacoplan and Other Currently Approved Complement Inhibitors

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and potentially life-threatening hematologic disorder caused by a somatic mutation in a relevant portion of hematopoietic stem cells. Mutation of the phosphatidylinositol glycan biosynthesis class A (PIGA) gene prevents the expression of cell-surfa...

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Bibliographic Details
Published in:	International journal of molecular sciences Vol. 25; no. 17; p. 9477
Main Authors:	Hillmen, Peter, Horneff, Regina, Yeh, Michael, Kolev, Martin, Deschatelets, Pascal
Format:	Journal Article
Language:	English
Published:	Switzerland MDPI AG 01.09.2024
Subjects:	Anemia Antibodies, Monoclonal, Humanized - pharmacology Antibodies, Monoclonal, Humanized - therapeutic use Blood Bone marrow CD55 Antigens - genetics CD55 Antigens - metabolism CD59 Antigens - genetics CD59 Antigens - metabolism Cloning Complement Activation - drug effects Complement Inactivating Agents - therapeutic use Disease FDA approval Hemoglobinuria, Paroxysmal - drug therapy Hemoglobinuria, Paroxysmal - metabolism Humans Immune system Laboratories Leukocytes Monoclonal antibodies Mortality Pathogens Proteins Stem cells Thrombosis C3 inhibitor paroxysmal nocturnal hemoglobinuria factor B inhibitor factor D inhibitor complement pegcetacoplan C5 inhibitor
ISSN:	1422-0067, 1661-6596, 1422-0067
Online Access:	Get full text
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