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- W4220774872 abstract "Abstract Approximately a third of patients with paroxysmal nocturnal hemoglobinuria (PNH) remain transfusion dependent or have symptomatic anemia despite treatment with a C5 inhibitor. Pegcetacoplan inhibits complement proximally at the level of C3 and is highly effective in treating persistent anemia resulting from C3-mediated extravascular hemolysis. We describe the rationale for C3 inhibition in the treatment of PNH and discuss preclinical and clinical studies using pegcetacoplan and other compstatin derivatives. We propose an approach for sequencing complement inhibitors in PNH." @default.
- W4220774872 created "2022-04-03" @default.
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- W4220774872 date "2022-06-09" @default.
- W4220774872 modified "2023-09-30" @default.
- W4220774872 title "Pegcetacoplan for paroxysmal nocturnal hemoglobinuria" @default.
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- W4220774872 doi "https://doi.org/10.1182/blood.2021014868" @default.
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