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Consensus on Complement Inhibitor Therapy and Management of Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH), a rare clonal disease of hematopoietic stem cells caused by mutations in the phosphatidylinositol N-acetylglucosaminyltransferase subunit A (PIGA) gene, may lead to the deficiency of glycosylated phosphatidylinositol (GPI)-anchored proteins. This deficiency...

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Autor principal: Red Blood Cell Disease (Anemia) Group, Chinese Society of Hematology, Chinese Medical Association
Format: Artigo
Idioma:Chinês
Publicat: Editorial Office of Journal of Rare Diseases 2025-01-01
Col·lecció:罕见病研究
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Accés en línia:https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2025.01.012
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