Genetic variants in C5 and poor response to eculizumab.

  title={Genetic variants in C5 and poor response to eculizumab.},
  author={Jun-ichi Nishimura and Masaki Yamamoto and Shin Hayashi and Kazuma Ohyashiki and Kiyoshi Ando and Andres L. Brodsky and Hideyoshi Noji and Kunio Kitamura and Tetsuya Eto and Toru Takahashi and Masayoshi Masuko and Takuro Matsumoto and Yuji Wano and Tsutomu Shichishima and Hirohiko Shibayama and Masakazu Hase and Lan Li and Krista Johnson and Alberto Lazarowski and Paul Tamburini and Johji Inazawa and Taroh Kinoshita and Yuzuru Kanakura},
  journal={The New England journal of medicine},
  volume={370 7},
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 or poor response to eculizumab. We also evaluated the functional properties of C5… CONTINUE READING


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