Genetic Variants in C5 and Poor Response to Eculizumab

Nishimura, Jun‐ichi; Yamamoto, Masaki; Hayashi, Shin; Ohyashiki, Kazuma; Ando, Kiyoshi; Brodsky, Andrés; Noji, Hideyoshi; Kitamura, Kunio; Eto, Tetsuya; TAKAHASHI, Toru; Masuko, Masayoshi; Matsumoto, Takuro; Wano, Yuji; Shichishima, Tsutomu; Shibayama, Hirohiko; Hase, Masakazu; Li, Lan; Johnson, Krista; Lazarowski, Alberto; P. Tamburini, Paul; Inazawa, Johji; Kinoshita, Taroh; Kanakura, Yuzuru

doi:10.1056/nejmoa1311084

Public

Genetic Variants in C5 and Poor Response to Eculizumab

Published in New England Journal of Medicine • Feb 12, 2014

Authors:

Jun‐ichi Nishimura

Masaki Yamamoto

Shin Hayashi

Abstract

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 patien...

Subject

Eculizumab

Paroxysmal nocturnal hemoglobinuria

Medicine

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