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Brodsky RA et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood 2008;111(4):1804-7. Abstract

Browett P et al. Vemircopan (ALXN2050) monotherapy in paroxysmal nocturnal haemoglobinuria: Interim data from phase 2 open-label proof-of-concept study. Br J Haematol 2023;201(S1). Abstract

Colden MA et al. Insights into the emergence of paroxysmal nocturnal hemoglobinuria. Frontiers Immunol 2022;12:830172. Abstract

de Latour RP et al. Oral monotherapy with iptacopan, a proximal complement inhibitor of factor b, has superior efficacy to intravenous terminal complement inhibition with standard of care eculizumab or ravulizumab and favorable safety in patients with paroxysmal nocturnal hemoglobinuria and residual anemia: Results from the randomized, active-comparator-controlled, open-label, multicenter, phase III Apply-PNH study. ASH 2022;Abstract LBA-2.

Hillmen P et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2006;355(12):1233-43. Abstract

Hillmen P et al. Effects of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 2004;350(6):552-9. Abstract

Lee JW et al. Patients with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis on ravulizumab/eculizumab showed hemoglobin response superiority with add-on danicopan vs placebo. EHA 2023;Abstract P771.

Liu H et al. Results from the first phase 3 crovalimab (C5 inhibitor) study (COMMODORE 3): Efficacy and safety in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria (PNH). Blood 2022;140(suppl 1):714-6. Abstract

Nishimura J-I et al. Mitigating drug-target-drug complexes in patients with paroxysmal nocturnal hemoglobinuria who switch C5 inhibitors. Clin Pharmacol Ther 2023;113(4):904-15. Abstract

Risitano AM et al. Oral iptacopan monotherapy has superior efficacy to anti-C5 therapy in patients with paroxysmal nocturnal hemoglobinuria and residual anemia: Results from the phase III APPLY-PNH study. EMBT 2023;Abstract OS12-01.

Roth A et al. The phase III, randomized COMMODORE 2 trial: Results from a multicenter study of crovalimab vs eculizumab in paroxysmal nocturnal hemoglobinuria (PNH) patients naïve to complement inhibitors. EHA 2023;Abstract S181.

Sheinberg P et al. Phase III randomized, multicenter, open-label COMMODORE 1 trial: Comparison of crovalimab vs eculizumab in complement inhibitor-experienced patients with paroxysmal nocturnal hemoglobinuria (PNH). EHA 2023;Abstract S183.