Real-world experience in management of patients with PNH treated with iptacopan: A case series. (PubMed, Blood Cells Mol Dis) - Oct 9, 2026 - "Notably, BTH did not occur in the patient with gastroenteritis, which can cause drug malabsorption. This case series suggests that iptacopan effectively maintains complement inhibition and prevents PNH-related hemolysis and thrombosis in the setting of past thromboses, gastrointestinal illness, and other CACs."
Journal • Real-world evidence • Cardiovascular • Complement-mediated Rare Disorders • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Otorhinolaryngology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Respiratory Diseases • Thrombosis
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Carmelo Gurnari; David Dingli; Jaroslaw P Maciejewski; Mark Orland; Srinivasa Sanikommu
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