Empaveli (pegcetacoplan) can ease disease burden for people with paroxysmal nocturnal hemoglobinuria (PNH), reducing signs of red blood cell destruction, lowering the need for blood transfusions, and improving fatigue and quality of life, according to a review of 12 studies conducted after Empaveli’s approval in 2021. “These findings, based…
Discussion
Nine years ago, I heard words that would completely change my life: I was diagnosed with paroxysmal nocturnal hemoglobinuria (PNH). At the time, I felt overwhelmed by fear and uncertainty. Like many people diagnosed with a rare disease, I had never heard of it before. Suddenly, I found myself…
A stem cell transplant using donor cells from close relatives was curative in people with paroxysmal nocturnal hemoglobinuria (PNH) co-occurring with aplastic anemia, a condition in which the bone marrow fails to produce enough blood cells, according to a study. At a median of nearly five years after the…
Discussion
On May 16, I had the opportunity to attend a patient and family conference in Tampa, Florida, with the Aplastic Anemia and MDS International Foundation, and I walked away with something much bigger than the usual information and resources. I left with connection, understanding, and a renewed sense of…
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