ArticleHaematologica2017
Small-molecule factor D inhibitors selectively block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Article in Haematologica, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 59 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
59 citing papers in PubMed, 98 citations in OpenAlex.
- Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.Blood advances · 2026Trial
- Trial
- Complement Inhibition in the Clinic: Are We Doing Enough to Protect Patients From Infection?European journal of immunology · 2026Review
- Shifting From Systemic to Precision-Targeted Complement Therapies: Opportunities and Hurdles.European journal of immunology · 2026Review
- Haemolytic Anaemia; Beyond Sickle Cell Anaemia: A Case Series of Patients With Paroxysmal Nocturnal Haemoglobinuria in Kenya.Case reports in hematology · 2026Article
- Integrative Landscape of Dry AMD Pathogenesis, Models, and Emerging Therapeutic Strategies.International journal of molecular sciences · 2025Review
- Paroxysmal Nocturnal Hemoglobinuria: Unraveling Its Molecular Pathogenesis and Advancing Targeted Therapeutic Strategies.Diseases (Basel, Switzerland) · 2025Review
- Highlights on U.S. FDA-approved halogen-containing drugs in 2024.European journal of medicinal chemistry · 2025Review
- Advances in Complement Inhibition Therapies for Paroxysmal Nocturnal Hemoglobinuria and Autoimmune Hemolytic Disorders.Journal of blood medicine · 2025Review
- Thrombosis in Paroxysmal Nocturnal Hemoglobinuria (PNH): From Pathogenesis to Treatment.International journal of molecular sciences · 2024Review
- The Complement System as a Therapeutic Target in Retinal Disease.Medicina (Kaunas, Lithuania) · 2024Review
- Complement inhibitors in pediatric kidney diseases: new therapeutic opportunities.Pediatric nephrology (Berlin, Germany) · 2024Review
- Role of complement factor D in cardiovascular and metabolic diseases.Frontiers in immunology · 2024Review
- Moss-produced human complement factor H with modified glycans has an extended half-life and improved biological activity.Frontiers in immunology · 2024Article
- The Role of Adipsin, Complement Factor D, in the Pathogenesis of Graves' Orbitopathy.Investigative ophthalmology & visual science · 2023Article
- Article
- Danicopan, an Oral Complement Factor D Inhibitor, Exhibits High and Sustained Exposure in Ocular Tissues in Preclinical Studies.Translational vision science & technology · 2022Article
- Recent Advances in Age-Related Macular Degeneration Therapies.Molecules (Basel, Switzerland) · 2022Review
- Complement-mediated microvascular injury and thrombosis in the pathogenesis of severe COVID-19: A review.World journal of experimental medicine · 2022Review
- Intertwined pathways of complement activation command the pathogenesis of lupus nephritis.Translational research : the journal of laboratory and clinical medicine · 2022Review
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
Abstract
Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome are diseases of excess activation of the alternative pathway of complement that are treated with eculizumab, a humanized monoclonal antibody against the terminal complement component C5. Eculizumab must be administered intravenously, and moreover some patients with paroxysmal nocturnal hemoglobinuria on eculizumab have symptomatic extravascular hemolysis, indicating an unmet need for additional therapeutic approaches. We report the activity of two novel small-molecule inhibitors of the alternative pathway component Factor D using
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.