Levels of certain immune proteins in the blood may help predict how well people with paroxysmal nocturnal hemoglobinuria (PNH) respond to the therapies Soliris (eculizumab) and PiaSky (crovalimab-akkz), a new study reports. “These findings indicate that higher baseline levels of these markers may help identify patients at…

When you live with paroxysmal nocturnal hemoglobinuria (PNH), you learn early on that no two days are ever the same. Some days, I wake up with energy and hope. Other days, I’m reminded that this rare disease is unpredictable — it can shift and change without warning. One of…

Vemircopan, a therapy that Alexion, AstraZeneca Rare Disease was developing for paroxysmal nocturnal hemoglobinuria (PNH), increased hemoglobin levels in a Phase 2 study, but frequent episodes of hemolysis — the destruction of red blood cells — were noted.  In a…