PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already receiving standard treatments but still experience significant red blood cell destruction. Participants must be on SOLIRIS or ULTOMIRIS for at least 6 months …

This study observes how well the drug iptacopan works for adults with paroxysmal nocturnal hemoglobinuria (PNH) during routine medical care in Russia. Researchers will follow 100 patients already prescribed iptacopan by their doctors to see if it increases their hemoglobin levels…

This study is testing a combination of two new drugs, pozelimab and cemdisiran, for people with PNH whose current standard treatment isn't fully controlling the disease. The goal is to see if this new combination can better reduce the destruction of red blood cells and improve sy…

This study is testing a new oral pill called HS-10542 for adults with PNH, a rare blood disease where the immune system destroys red blood cells. The goal is to see if the pill can safely control the disease and raise hemoglobin levels in people who still have active destruction …

This Phase 3 study is testing the long-term safety and effectiveness of a two-drug combination (pozelimab + cemdisiran) for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare and serious blood disease. The trial will follow 202 patients who previously participated in r…

This study is monitoring the safety and effectiveness of Fabhalta capsules in 100 patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track patients for 48 weeks after starting treatment to watch for infections, side effects, and how w…

This study is testing whether adding a new drug called danicopan helps children with a rare blood disease called PNH. The children are already on standard treatment but still have significant anemia. The main goal is to see if the add-on drug increases their hemoglobin levels (a …

This study is observing how a drug called iptacopan affects adults in China with a rare blood disease called paroxysmal nocturnal hemoglobinuria (PNH). It will follow 80 patients who are starting this new treatment to see if it improves their blood counts, reduces symptoms, and c…

This study is testing a new combination of two drugs, pozelimab and cemdisiran, for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. The main goal is to see if this new combination is more effective and safe than two existing standard treatments, ravul…