Disorder of glycosylation

This study tests a new drug called NM8074 in 12 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking Soliris. PNH is a rare blood disorder where the immune system destroys red blood cells, causing anemia and fatigue. NM8074 is designed to block a different…

This phase II trial tests the drug SLN12140 in 10 adults with PNH who have never taken complement inhibitors. The goal is to see if SLN12140 can reduce red blood cell destruction (measured by LDH levels) and improve hemoglobin. Participants receive different doses over 12 to 64 w…

This study looks at the long-term safety and effectiveness of an injection called LP-005 in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It is for people who already completed an earlier LP-005 study. The goal is to see if LP-005 can help control …

This Phase 3 trial tests NTQ5082, an oral capsule, against the standard infusion eculizumab for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 78 adults who have not had complement inhibitor therapy will be enrolled. The main goal is to see if NTQ5082 can…

This Phase 3 trial tests NTQ5082 capsules, a new drug that blocks a protein called CFB, in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study will follow 78 adults who previously benefited from NTQ5082 or took ano…