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Hemasphere|June 8, 2026
Iptacopan monotherapy resulted in increased hemoglobin level in patients with PNH and hemoglobin ≥10 g/dL after anti-C5 therapyAustin Kulasekararaj, Flore S de Fontbrune, Anna Gaya, et al.The New England Journal of Medicine|March 13, 2024
Oral Iptacopan Monotherapy in Paroxysmal Nocturnal HemoglobinuriaRégis Peffault de Latour, Alexander Röth, Austin G Kulasekararaj, et al.The Lancet. Haematology|May 30, 2025
Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitorsAntonio M Risitano, Austin G Kulasekararaj, Phillip Scheinberg, et al.Pageof 4