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…
News
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…
Empaveli (pegcetacoplan) was safely administered during a woman’s pregnancy to manage hard-to-control paroxysmal nocturnal hemoglobinuria (PNH), according to a recent study. Results showed that Empaveli effectively controlled the mother’s PNH, and the baby was born without complications. There were no detectable signs of the medication in…
Doctors for the first time used PiaSky (crovalimab-akkz) to control paroxysmal nocturnal hemoglobinuria (PNH) in a woman with the autoimmune disease systemic lupus erythematosus (SLE), according to a case study. The study’s authors said the case is the seventh documented instance of PNH occurring in someone with…
A case report highlights the steps necessary to prevent dangerous clots and minimize bleeding when performing a cesarean section on someone who has co-occurring paroxysmal nocturnal hemoglobinuria (PNH) and aplastic anemia, a condition in which the bone marrow fails to produce enough blood cells. Researchers in Greece described the…
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…
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…
Recent Posts
- Rethinking what control looks like after a PNH diagnosis
- New study backs approved Voydeya dosing for PNH add-on treatment
- In the rare disease community, we find strength in each other
- Complement biomarkers may not reliably track PNH disease activity
- Improving quality of life is key to living fully with PNH