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In a woman with paroxysmal nocturnal hemoglobinuria (PNH) who developed acute kidney damage and low numbers of red blood cells and platelets during a COVID-19 infection, doctors suspected another disease called complement-mediated thrombotic microangiopathy (TMA). Her condition rapidly improved after treatment with Soliris (eculizumab). “Viral insults can serve…

Seven people with paroxysmal nocturnal hemoglobinuria (PNH) in Kenya went months to years before receiving a diagnosis, according to a case series highlighting the challenges of recognizing and treating the rare, acquired blood disorder in resource-limited settings. All seven had recurrent anemia, or a shortage of healthy red blood…

Anemia, or a shortage of healthy red blood cells, may have a greater impact on quality of life than ongoing red blood cell destruction — the hallmark of paroxysmal nocturnal hemoglobinuria (PNH) — despite treatment in people with the rare acquired disease, a real-world study suggests. Individuals with significant…

Use of an experimental infusion therapy for paroxysmal nocturnal hemoglobinuria (PNH) helped increase the levels of hemoglobin — the oxygen-carrying protein in red blood cells — while reducing the need for blood transfusions among participants in a small trial. Data show the mid-stage clinical trial testing HSK39297, in the…

Jasper Therapeutics has announced its acquisition of Kira Pharmaceuticals in a deal that will provide new resources and expertise to help advance the late-stage clinical development of KP104, a treatment candidate for paroxysmal nocturnal hemoglobinuria (PNH), as well as other experimental therapies involving the immune system. For…

While people with paroxysmal nocturnal hemoglobinuria (PNH) have access to a range of approved medications that effectively control the rare acquired disease, their administration can be time-consuming and may disrupt patients’ work, travel, daily activities, and family life, creating a large time burden. That’s according to a new study…

Use of the approved injection therapy Empaveli (pegcetacoplan) appears to be effective and generally safe for people with paroxysmal nocturnal hemoglobinuria (PNH) — especially for patients who, despite treatments, have ongoing destruction of red blood cells, known as hemolysis. Those are the findings of a new analysis from…

Treatment with Soliris (eculizumab) appears to help reduce the risk of potentially life-threatening thromboembolic events (TEs), complications caused by clots that disrupt circulation, in people with paroxysmal nocturnal hemoglobinuria (PNH), but it does not eliminate that risk, according to a post-marketing surveillance study from Japan. Researchers found that…

Most adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from standard C5 inhibitors to Fabhalta (iptacopan) saw their hemoglobin levels rise, remained free from blood transfusions, and reported less fatigue after six months of treatment, according to Phase 3 trial results. The open-label Phase 3b APPULSE-PNH study (NCT05630001) was designed…

Extensive clotting in the skin’s small blood vessels, known as dermal thrombosis, was identified in a 44-year-old man who suffered multiple strokes and was later diagnosed with paroxysmal nocturnal hemoglobinuria (PNH), according to a case report from India. Researchers noted that “prompt recognition of this unusual PNH-related skin thrombosis…