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Articles

Clinical Experience of Iptacopan in Paroxysmal Nocturnal Hemoglobinuria

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By Matthew Holt, Richard Kelly, LIMR Leeds, Richard Kelly, Regis de Latour, Morag Griffin, Shreyans Gandhi, Edouard Forcade, Francesco Versino, Roochi Trikha, Alexandra C Pike, Joanna Large, Michael Loschi, Marc Michel, Petra Muus, Talha Munir, Alice Garnier
| Wiley Online Library Verified
1 Introduction Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare disorder of hematopoietic stem cells, characterized by chronic complement-mediated intravascular hemolysis (IVH) and a predisposition to thrombosis [1]. Standard management of symptomatic PNH in the UK and France is with complement inhibition.
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