Omeros’ zaltenibart (OMS906) as a single therapy has shown promise as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) in a small clinical study, normalizing signs of hemolysis, or red blood cell destruction, in adults with hard-to-treat disease, according to new interim data. The trial enrolled PNH patients who…
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What does it mean to “live rare”? After I was diagnosed with two rare blood disorders, aplastic anemia and paroxysmal nocturnal hemoglobinuria (PNH), it took me years to understand how to navigate tough times and adapt to life with my rare diseases. According to the National Organization…
Treatment with Ultomiris (ravulizumab) successfully reduced multiple blood clots that didn’t respond to standard anti-clotting therapy in a woman with paroxysmal nocturnal hemoglobinuria (PNH), a case study shows. “This report presents a case of myocardial and renal infarction [heart and kidney tissue death due to blood supply obstruction]…
England’s National Institute for Health and Care Excellence (NICE) has recommended that the oral treatment Voydeya (danicopan) be covered by the country’s National Health Service (NHS) and provided at low or no cost to certain adults with paroxysmal nocturnal hemoglobinuria (PNH). The recommendation covers Voydeya’s use as an…
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