Iptacopan monotherapy demonstrated improved clinical outcomes in a real-world cohort with paroxysmal nocturnal hemoglobinuria: Evidence from a managed access program
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DOI: 10.1182/blood-2025-3202
Abstract: Iptacopan, the first-in-class oral complement factor B inhibitor that selectively inhibits the alternative pathway of the complement system, has been approved for the treatment (tx) of patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH). In 2023,… read more here.
Keywords: rr1 rr2; paroxysmal nocturnal; iptacopan; program ... See more keywords