Blood markers designed to measure activation of the complement system, a part of the immune system involved in paroxysmal nocturnal hemoglobinuria (PNH), may not reliably reflect disease activity or red blood cell destruction in patients receiving complement-blocking therapies, a new study suggests. The researchers found that although these complement…
Discussion
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,…
Get regular updates delivered to your inbox.
