Antonio Risitano, MD, PhD, highlights exciting developments in treatments for paroxysmal nocturnal hemoglobinuria (PNH), specifically the introduction of complement inhibitors that have transformed the prognosis of the disease. A new oral drug, iptacopan, shows promising results, offering nearly normal hemoglobin levels and reducing the need for transfusions in PNH patients. Data from recent studies confirm sustained efficacy and low rates of breakthrough hemolysis. The outlook for PNH patients continues to improve, with the potential for better quality of life and disease management.