February 29 is International Rare Disease Day. PNH stands for paroxysmal nocturnal hemoglobinuria.
Patients often suffer from severe anemia, blood clots, and kidney function damage. The advent of C5 complement inhibitors represented by eculizumab has changed the natural course of the disease, allowing patients to live closer to normal while improving their quality of life. But there are still many practical problems that need to be solved, such as transportation issues and medical insurance reimbursement issues in other places.