Paroxysmal nocturnal hemoglobinuriaNews & Research
16 curated articles for Paroxysmal nocturnal hemoglobinuria — FDA updates, peer-reviewed research, clinical-trial milestones, and sponsor press releases. Sorted newest-first.
- Leukemia Apr 22, 2026
Imputing causality and clonal dynamics from single-cell transcriptomics in paroxysmal nocturnal hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (PNH) originates from hematopoietic stem cells (HSCs) harboring somatic mutations in the phosphatidylinositol glycan class A (PIGA) gene. Clonal expansion of PIGA-mutated cells occurs uniquely in the setting of bone marrow (BM) failure, but specific pathophysiolog...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- Nefrologia Apr 17, 2026
Complement blockade: Therapeutic promises and remaining challenges in clinical practice.
Understanding of the complement system in glomerulopathies has advanced significantly, revealing that this system-beyond its role in innate immunity-is a key mediator of kidney injury across a wide spectrum of nephropathies, from those driven by immune complexes to those caused by primary dysregulat...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- PloS one Apr 16, 2026
Epidemiology and care management of Paroxysmal Nocturnal Hemoglobinuria (PNH) in a real-world setting in France: Description from the French National Hospitalization Database.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired stem-cell disease causing anemia, aplasia and thromboses, affecting ~1/80,000 patients in France. Treatment mainly involves C5 inhibitors (C5i) and iterative red-blood-cell transfusions. Recent real-world French data on PNH epidemiology a...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- Blood advances Apr 15, 2026
Ravulizumab for Treatment of Paroxysmal Nocturnal Hemoglobinuria During Pregnancy.
Ravulizumab, a humanized, long-acting monoclonal antibody against complement factor C5, is a widely utilized treatment for paroxysmal nocturnal hemoglobinuria (PNH). As pregnant women with PNH are at increased risk for complications and the safety and efficacy of ravulizumab in pregnancy is not know...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- Annals of African medicine Apr 7, 2026
Acute Pancreatitis as a Therapeutic Complication in Case of Paroxysmal Nocturnal Hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired hematopoietic disorder requiring careful therapeutic management. In resource-limited settings, traditional treatments may carry significant risks. A 32-year-old female with PNH developed acute pancreatitis 10 months after initiation of dan...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- Hematology (Amsterdam, Netherlands) Mar 20, 2026
Case report: persistent anemia after eculizumab in paroxysmal nocturnal hemoglobinuria: non-dominantly active intravascular hemolysis.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare condition characterized by intravascular hemolysis (IVH), thrombosis, and organ damage. Although C5 complement inhibitors such as eculizumab have significantly improved the prognosis of patients with PNH, persistent anemia remains in some patients....
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- American journal of hematology Mar 17, 2026
Complement Biomarkers for Monitoring Last-Generation Complement Inhibitors in Paroxysmal Nocturnal Hemoglobinuria Patients.
Complement Biomarkers for Monitoring Last-Generation Complement Inhibitors in Paroxysmal Nocturnal Hemoglobinuria Patients.
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- Kidney international Mar 13, 2026
Renal hemosiderosis in paroxysmal nocturnal hemoglobinuria.
Renal hemosiderosis in paroxysmal nocturnal hemoglobinuria.
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- European journal of haematology Mar 12, 2026
Allogeneic Hematopoietic Cell Transplantation Using Post-Transplant Cyclophosphamide in Patients With Large Paroxysmal Nocturnal Hemoglobinuria Clones.
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for paroxysmal nocturnal hemoglobinuria (PNH). Post-transplant cyclophosphamide (PTCy) has improved HSCT safety in other diseases, but its use in PNH is poorly characterized. In this retrospective study, we anal...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- CPT: pharmacometrics & systems pharmacology Mar 6, 2026
Population PK-PD Modeling of Danicopan Add-On Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria Treated With Ravulizumab or Eculizumab.
Danicopan is a first-in-class orally administered complement factor D inhibitor, approved as an add-on therapy to ravulizumab or eculizumab for the treatment of clinically significant extravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH). Population pharmacokinetics (PK)...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- Journal of patient-reported outcomes Feb 20, 2026
Psychometric performance of the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue questionnaire among adults with paroxysmal nocturnal hemoglobinuria.
Two Phase 3 clinical trials were conducted to investigate the efficacy and safety of iptacopan, a novel treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hemolytic disorder whose most prevalent symptom is fatigue. The FACIT-Fatigue questionnaire version 4 was administered to p...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- Nephrologie & therapeutique Feb 17, 2026
[Targeting complement in atypical hemolytic and uremic syndrome: development and current status of eculizumab and ravulizumab].
Until 2009, atypical hemolytic uremic syndrome (aHUS), a disease mediated by complement-induced endothelial activation, had a poor renal prognosis, with common progression to kidney failure and frequent recurrence after kidney transplantation, condemning often young patients to lifelong dialysis. In...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- International journal of hematology Feb 8, 2026
Clonal expansion mechanisms in paroxysmal nocturnal hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by complement-dependent intravascular hemolysis and thrombosis as well as bone marrow failure. Complement dysregulation occurs as a result of defective cell surface expression of two glycosylphosphatidylinositol (GPI)-anchored complement reg...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- Blood advances Jan 28, 2026
Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, complement-mediated hematologic disease. We present the results of a phase 2, open-label clinical trial of vemircopan monotherapy in patients with PNH to explore the efficacy and safety of inhibiting the complement alternative pathway via factor D...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- Current opinion in immunology Jan 15, 2026
Complement-targeting therapies in hemolytic diseases.
Complement inhibition has revolutionized the management of hemolytic diseases by targeting the underlying drivers of red blood cell destruction in disorders such as paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and cold agglutinin disease. Recent advances have expande...
Why it matters: Recent peer-reviewed research on Paroxysmal nocturnal hemoglobinuria that may be relevant for patients and caregivers.
- BreakingFDA Aug 30, 2025
PNH Patients Gain Subcutaneous Complement Inhibitor
New subcutaneous complement C5 inhibitor approved for PNH, offering self-administered alternative.
Why it matters: Home-based treatment reduces hospital visits for PNH patients.
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Articles aggregated from peer-reviewed journals (PubMed), sponsor press releases, SEC 8-K filings, and FDA announcements. Original-source links are preserved on each article page. Editorial tags (Breaking / Notable / Update) reflect UniteRare's curation-time priority assessment.