Blood advancesHumansHemoglobinuriaParoxysmalMaleFemale
Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.
Austin Kulasekararaj, Peter Browett, Antonio M Risitano, Christopher J Patriquin, Mustafa N Yenerel, Daniele Marceau, Fahri Sahin, J Lorenzo Algarra, Masayo Ogawa, Ji Yu, Nicholas Bruce Cross, Rosario Notaro, Jong Wook Lee, Robert A Brodsky
Published: 202610.1182/bloodadvances.2025017731
Abstract
ABSTRACT: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, complement-mediated hematologic disease. We present the results of a phase 2, open-label clinical trial of vemircopan monotherapy in patients with PNH to explore the efficacy and safety o…
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