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: | |
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| Formato: | Artigo |
| Idioma: | Chinês |
| Publicado em: |
Editorial Office of Journal of Rare Diseases
2025-01-01
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| coleção: | 罕见病研究 |
| Assuntos: | |
| Acesso em linha: | https://jrd.chard.org.cn/article/doi/10.12376/j.issn.2097-0501.2025.01.012 |
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