Living with paroxysmal nocturnal hemoglobinuria (PNH) changes many things, but one of the most meaningful shifts happening in the PNH community today is the growing focus on quality of life. For years, conversations about rare diseases focused mainly on survival and symptom management. Today, patients, doctors, and advocates…
The first clinical report of a patient with paroxysmal nocturnal hemoglobinuria (PNH) switching from PiaSky (crovalimab-akkz) to Fabhalta (iptacopan) was reported recently. The patient in the report was a 72-year-old man who continued to experience PNH symptoms after an infection triggered…
Antibody-based therapy ruxoprubart (formerly NM8074) safely prevented blood cell destruction, eased anemia, and eliminated the need for blood transfusions when given as a standalone treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to results from a Phase 2 trial (NCT05646524) announced by its developer,…
Use of the approved therapy Ultomiris (ravulizumab) keeps hemolysis, or the destruction of red blood cells, under control, reduces the need for transfusions, and is generally well tolerated for people with paroxysmal nocturnal hemoglobinuria (PNH), a real-world study in Italy has found. According to the researchers, the benefits…
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