The long-term efficacy and safety of iptacopan in treating Paroxysmal Nocturnal Hemoglobinuria (PNH) with anaemia was a key topic at ASH 2023. Prof. Antonio Risitano from the University of Naples, Italy, presented findings that highlight the potential of iptacopan as a treatment option for patients suffering from this condition. PNH is a rare, life-threatening blood disorder characterized by the destruction of red blood cells, leading to anaemia and other complications. The presentation underscored the importance of ongoing research and clinical trials to evaluate the effectiveness and safety of new treatments like iptacopan in improving the quality of life for patients with PNH.