Efficacy and safety of HRS-5965 monotherapy in complement inhibitor-naïve patients with paroxysmal nocturnal haemoglobinuria.

Summary: Imagine your body's defense system accidentally attacking your own red blood cells. That is what happens in a rare disease called PNH, leaving people very tired and sick. A new daily pill called HRS-5965 might be able to help. In a recent study, 26 patients took this pill for 12 weeks. The results were great! The pill stopped the blood cells from breaking apart, and the patients' healthy red blood cell levels went way up. The side effects were mild, making this new pill a very promising treatment for people with PNH.