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Coverage: 2020-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
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Paroxysmal Nocturnal Haemoglobinuria: Research Summary
Printed from RareWays (rareways.org)
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Ciprocopan: First Approval.
Fung Simon, Drugs (15 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42875656/
- 2.
Safety of Ravulizumab Use in Pregnancy: Insights from a Global Pharmacovigilance Analysis.
Carrillo Infante Cynthia et al., Advances in therapy (9 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42853533/
- 3.
AI-assisted recognition of misdiagnosed paroxysmal nocturnal hemoglobinuria
Adam Bowen et al., Orphanet Journal of Rare Diseases (5 October 2026)
https://doi.org/10.1186/s13023-026-04629-3
- 4.
Multiparametric Ultrasound in Budd-Chiari Syndrome: From Venous Outflow Obstruction to Microcirculatory Dysfunction.
Fedak Andrzej et al., Journal of ultrasound in medicine : official journal of the American Institute of Ultrasound in Medicine (3 October 2026)
https://pubmed.ncbi.nlm.nih.gov/42828434/
- 5.
Real-world effectiveness of pegcetacoplan in individuals with paroxysmal nocturnal haemoglobinuria and suboptimal response to C5 inhibitors
Łukasz Szukalski et al., Therapeutic Advances in Hematology (1 October 2026)
https://doi.org/10.1177/20406207261495852
Clinical Trials : Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials. Each has its ID and full eligibility criteria at the link shown.
- 1.
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
NCT05876312 · Recruiting · Phase 1 / Phase 2 · ADARx Pharmaceuticals, Inc.
Ages 18 and over
https://clinicaltrials.gov/study/NCT05876312
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Paroxysmal Nocturnal Haemoglobinuria
Paroxysmal Nocturnal Haemoglobinuria is a rare acquired disorder of haematopoietic stem cells caused by a somatic mutation in the PIG-A gene, leading to complement-mediated destruction of red blood cells. It causes haemolytic anaemia, thrombosis, and bone marrow failure. Complement inhibitors (eculizumab, ravulizumab) are transformative treatments that dramatically reduce complications.
This overview is general information, not an individual medical assessment. Research is selected automatically and may include mismatches. How we select and explain research.
Most Recent Research
Ciprocopan (®) is a small molecule inhibitor of complement factor B, which is a key component of the alternative complement pathway. It is being developed by Haisco Pharmaceutical Group for the treatment of complement-mediated disorders. In July 2026, ciprocopan received its first approval for treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have not previously received treatment with a complement inhibitor therapy in China. This article summarizes the milestones in the development of ciprocopan leading to this first approval.
Common Questions
What is Paroxysmal Nocturnal Haemoglobinuria?
Paroxysmal Nocturnal Haemoglobinuria is a rare acquired disorder of haematopoietic stem cells caused by a somatic mutation in the PIG-A gene, leading to complement-mediated destruction of red blood cells. It causes haemolytic anaemia, thrombosis, and bone marrow failure. Complement inhibitors (eculizumab, ravulizumab) are transformative treatments that dramatically reduce complications.
How many clinical trials are available for Paroxysmal Nocturnal Haemoglobinuria?
RareWays currently indexes 129 clinical trials for Paroxysmal Nocturnal Haemoglobinuria, of which 16 are actively recruiting. These counts cover indexed trials across countries, not just your country. An Australian site does not necessarily mean that site is recruiting. Check locations, eligibility and current availability with the study team.
Where does the research data for Paroxysmal Nocturnal Haemoglobinuria come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
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