Paroxysmal nocturnal haemoglobinuria: targeting C3 delivers better outcomes

Pegcetacoplan offered better haemtologic and clinical outcomes than eculizumab in a UK trial of patients with paroxysmal nocturnal haemoglobinuria (PNH). The therapy, which targets complement C3  could offer an option to patients who experience persistent anaemia despite long-term therapy with C5 inhibitors. PNH is a rare acquired disease, characterised by complement-mediated haemolysis. Patients often suffer ...

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