News

Screening important for co-occurring PNH, aplastic anemia: Case study

Early screening for paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia — two bone marrow disorders that often occur together — is important to prevent acute and serious complications, according to a recent case study. Aplastic anemia affects the stem cells in the bone marrow that give rise to new blood…

Empaveli most effective in newly treated PNH patients in study

People with paroxysmal nocturnal hemoglobinuria (PNH) newly treated with Empaveli (pegcetacoplan) are more likely to see their hemoglobin levels rise and less likely to experience breakthrough disease attacks than those started on Soliris (eculizumab) or Ultomiris (ravulizumab-cwvz). That’s according to a new study reporting the findings from an indirect comparison…

Trial of PNH treatment ARO-C3 plans to start dosing patients this year

ARO-C3, an experimental treatment being developed for paroxysmal nocturnal hemoglobinuria (PNH) and other disorders mediated by the complement system, appears to be working as intended in a group of healthy volunteers. That’s according to interim data from an ongoing Phase 1/2 trial testing the Arrowhead Pharmaceuticals therapy in adults…

Soliris effective in PNH when properly prescribed: Study

Most people with paroxysmal nocturnal hemoglobinuria (PNH) benefited from Soliris (eculizumab) therapy when they adhered to strict treatment guidelines, a real-world Dutch study showed. Because most had an incomplete response, new therapies are needed to improve real-world outcomes, however, the study’s researchers suggested, and emphasized that their data could…