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Paroxysmal nocturnal hemoglobinuria, or PNH, is a rare disease characterized by hemolysis, thrombosis, and bone marrow failure. Intravenous anti-C5 monoclonal antibodies are the standard treatment; however, many patients remain anemic, mainly because of ongoing activation of extravascular hemolysis mediated by macrophages ingesting C3 fragment-coated red cells. Now, two phase 3 trials have evaluated the efficacy and safety of iptacopan, an oral complement factor B inhibitor, in PNH.    Research findings are summarized in a new Quick Take video. https://lnkd.in/eZAWEA46    Read the related Original Article by Régis Peffault de Latour, M.D., et al.: https://nej.md/48QKlcU    #ClinicalTrials #MedicalResearch 

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