A new 13-item questionnaire designed to assess fatigue and its impact on daily functioning in individuals with chronic illnesses appears to be accurate and may be appropriate for use in adults with paroxysmal nocturnal hemoglobinuria (PNH). That’s according to a new analysis evaluating the validated tool, dubbed the Functional Assessment…
News
Mutations in the MUC4 gene may be linked to a higher risk of dangerous blood clots in people with paroxysmal nocturnal hemoglobinuria (PNH), a study has found. Researchers believe the mutations may be associated with increased activity in part of the immune system called the complement system. In mouse…
Ultomiris (ravulizumab-cwvz) may be safe and effective as a treatment for paroxysmal nocturnal hemoglobinuria (PNH) in pregnant women, a study suggested. Of 19 pregnancies in women treated with Ultomiris, all resulted in live births, compared with four live births out of eight pregnancies previously managed with Soliris…
Early recognition of paroxysmal nocturnal hemoglobinuria (PNH) in a 39-year-old woman with unexplained anemia led to treatment starting before any blood clot, or thrombotic, complications were detected, according to a case report from Portugal. The woman sought emergency care multiple times after several weeks of worsening fatigue and recurrent…
Improved access to standard-of-care treatments could significantly improve outcomes for people with paroxysmal nocturnal hemoglobinuria (PNH) living in South India, a study found. The researchers said poor outcomes, including frequent complications, repeated hospitalizations, and increased mortality, were largely driven by greater reliance on supportive care in the region. “Supportive…
People with paroxysmal nocturnal hemoglobinuria (PNH) have abnormalities in infection-fighting immune cells called natural killer (NK) cells, a study showed, shedding light on how this blood cell disorder may dysregulate the immune system. The study, “Role of NK cells in immune escape in patients with classical…
A healthy baby was born after exposure to Ultomiris (ravulizumab-cwvz) during the first trimester of pregnancy in a 25-year-old woman with paroxysmal nocturnal hemoglobinuria (PNH), according to a new case report. The woman was on Ultomiris when she was found to be pregnant. Because little is known about…
In 2025, PNH News delivered timely coverage of the latest research developments and advances in care related to paroxysmal nocturnal hemoglobinuria (PNH). To close the year, we’ve compiled the five most-read stories. As we move into 2026, we remain committed to supporting the PNH community with clear reporting…
A rare case of paroxysmal nocturnal hemoglobinuria (PNH) coexisting with diffuse large B-cell lymphoma (DLBCL), a type of blood cancer, was chronicled in a new report that sought to elucidate some of the challenges that can come with the management of the acquired blood disorder co-occurring with this cancer.
Treatment with PiaSky (crovalimab-akkz) controls hemolysis — the destruction of red blood cells in the body — in nearly all people with paroxysmal nocturnal hemoglobinuria (PNH), according to the results of a pooled analysis of published clinical trials. Further, the data also showed that about two-thirds of individuals…
Recent Posts
- New questionnaire assessing fatigue ‘reliable and valid’ for adults with PNH
- Progress in research and treatments is a reason for holding on to hope
- New gene study highlights clot risk factor in people living with PNH
- Becoming a patient advocate gave me purpose in the fight against PNH
- Study finds Ultomiris safe as PNH treatment during pregnancy