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Article

Genetic Variants in C5 and Poor Response to Eculizumab

12 Feb 2014

Abstract excerpt

BACKGROUND: Eculizumab is a humanized monoclonal antibody that targets complement protein C5 and inhibits terminal complement-mediated hemolysis associated with paroxysmal nocturnal hemoglobinuria (PNH). The molecular basis for the poor response to eculizumab in a small population of Japanese patients is unclear. METHODS: We assessed the sequences of the gene encoding C5 in patients with PNH who had either a good...

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PMID: 24521109
DOI: 10.1056/nejmoa1311084

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