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3 weeks ago | 
pnhnews.com | Patricia Inacio |Katherine Poinsatte |Andrea Lobo |Brandi Lewis
Paroxysmal nocturnal hemoglobinuria (PNH) was identified as the likely cause of a stroke in a young man, according to a report from India.
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1 month ago | 
pnhnews.com | Margarida Maia |Brandi Lewis |Marisa Wexler MS |Steve Bryson
The use of Fabhalta (iptacopan) — approved in the U.S. in December 2023 as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) — may save about $4 million over a patient’s lifetime compared with intravenous, or into-the-vein, standard of care therapies, according to a study by U.S. researchers. The oral therapy was found, in the clinical studies that served as the basis for its U.S. approval, to be more convenient for patients than the use of other complement inhibitors for treating PNH.
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2 months ago | 
pnhnews.com | Margarida Maia |Steve Bryson |Marisa Wexler MS |Brandi Lewis
Most Chinese adults and adolescents with paroxysmal nocturnal hemoglobinuria (PNH) no longer require blood transfusions after a median of six months of treatment with Soliris (eculizumab). That’s according to a small study at a single center in China, which also found the approved therapy to be safe and well tolerated.
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2 months ago | 
pnhnews.com | Marisa Wexler MS |Esteban Cerezo |Brandi Lewis |Patricia Inacio
Long-term treatment with Ultomiris (ravulizumab) reduced the risk of death by five times in people with paroxysmal nocturnal hemoglobinuria (PNH), and also tended to eliminate the need for blood transfusions and to improve patients’ quality of life, a new analysis suggests.
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2 months ago | 
pnhnews.com | Margarida Maia |Brandi Lewis |Steve Bryson
Fatigue affects the ability of adults with paroxysmal nocturnal hemoglobinuria (PNH) to perform daily tasks, interact socially, and stay focused, even with treatment, showing how the disease limits physical, emotional, and social well-being, according to a real-world study.
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Jan 9, 2025 | 
oncnursingnews.com | David Dingli |Brandi Lewis
Panelists discuss how to help patients weigh the risks and benefits of different treatment options for paroxysmal nocturnal hemoglobinuria (PNH), outlining tools and techniques used to ensure that patients and caregivers fully understand their choices and make informed decisions.
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Dec 9, 2024 | 
pnhnews.com | Steve Bryson |Brandi Lewis |Andrea Lobo
Six months of twice-daily treatment with oral Fabhalta (iptacopan) improved hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from standard anti-C5 therapies, according to top-line results from a Phase 3 clinical trial. Dubbed APPULSE-PNH (NCT05630001), the now-complete study tested Novartis’ therapy in 52 adults with PNH.
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Oct 28, 2024 | 
pnhnews.com | Steve Bryson |Andrea Lobo |Brandi Lewis |Marisa Wexler MS
England’s National Institute for Health and Care Excellence (NICE) has recommended that the oral treatment Voydeya (danicopan) be covered by the country’s National Health Service (NHS) and provided at low or no cost to certain adults with paroxysmal nocturnal hemoglobinuria (PNH).
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Oct 21, 2024 | 
pnhnews.com | Marisa Wexler MS |Brandi Lewis |Andrea Lobo |Patricia Inacio
The cases of three people with paroxysmal nocturnal hemoglobinuria (PNH) who experienced serious complications after switching from an experimental medicine to the approved PNH treatment Ultomiris (ravulizumab) highlight the potential for serious risks when switching from a PNH treatment targeting earlier steps of complement activation to one targeting later steps, researchers said.
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Oct 7, 2024 | 
pnhnews.com | Patricia Inacio |Brandi Lewis |Erin Fortin
In rare cases, acute kidney injury may be sign of paroxysmal nocturnal hemoglobinuria (PNH), as described in a recent case report. “Although renal dysfunction in PNH is rare and as renal dysfunction is a dreadful complication, timely intervention is life-saving for the patient who requires hemodialysis,” the scientists wrote. The report, “Paroxysmal nocturnal hemoglobinuria presenting as acute renal injury,” was published in the International Journal of Research in Medical Sciences.
