ReviewClinical immunology (Orlando, Fla.)2015
Applying complement therapeutics to rare diseases.
Review in Clinical immunology (Orlando, Fla.), 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03316521 (Safety, Tolerability, Pharmacokinetics), which is not on this map. Cited by 33 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of a Single Ascending Dose (SAD) and a Multiple Dose (MD) of the Complement Inhibitor AMY-101. A Prospective, Single-center, Open-label, First-In-Human (FIH) Clinical Study in Healthy Male Volunteers
Who cites it
33 citing papers in PubMed, 60 citations in OpenAlex.
- Complement C3 deficiency enhances renal leptospiral load and inflammation while impairing T cell differentiation during chronicInfection and immunity · 2025Article
- Combinatorial activation of the WNT-dependent fibrogenic program by distinct complement subunits in dystrophic muscle.EMBO molecular medicine · 2023Article
- The role of complement C3 in the outcome of regional myocardial infarction.Biochemistry and biophysics reports · 2023Article
- Aptamers: Potential Diagnostic and Therapeutic Agents for Blood Diseases.Molecules (Basel, Switzerland) · 2022Review
- Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab.PloS one · 2021Article
- Editorial: Complement and Immunotherapeutics.Frontiers in immunology · 2021Article
- Alternative Complement Pathway Inhibition Does Not Abrogate Meningococcal Killing by Serum of Vaccinated Individuals.Frontiers in immunology · 2021Article
- Article
- Promising and Upcoming Treatments in Myositis.Current rheumatology reports · 2020Review
- Computational analysis of complement inhibitor compstatin using molecular dynamics.Journal of molecular modeling · 2020Article
- The first case of COVID-19 treated with the complement C3 inhibitor AMY-101.Clinical immunology (Orlando, Fla.) · 2020Article
- An inhibitor of complement C5 provides structural insights into activation.Proceedings of the National Academy of Sciences of the United States of America · 2020Article
- Podocytes Produce and Secrete Functional Complement C3 and Complement Factor H.Frontiers in immunology · 2020Article
- The complement system in liver diseases: Evidence-based approach and therapeutic options.Journal of translational autoimmunity · 2019Review
- Eidikology: proposition for a new terminology for the science of rare diseases.Postgraduate medical journal · 2019Article
- Invasive meningococcal disease in patients with complement deficiencies: a case series (2008-2017).BMC infectious diseases · 2019Article
- Expanding Complement Therapeutics for the Treatment of Paroxysmal Nocturnal Hemoglobinuria.Seminars in hematology · 2018Review
- Immunopathological Characterization of Muscle Biopsy Samples from Immune-Mediated Necrotizing Myopathy Patients.Medical science monitor : international medical journal of experimental and clinical research · 2018Article
- Expanding the therapeutic options for renal involvement in lupus: eculizumab, available evidence.Rheumatology international · 2017Review
- Complement C3-Targeted Therapy: Replacing Long-Held Assertions with Evidence-Based Discovery.Trends in immunology · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 3 institutions in 3 countries.
Funding
Abstract
Around 350 million people worldwide suffer from rare diseases. These may have a genetic, infectious, or autoimmune basis, and several include an inflammatory component. Launching of effective treatments can be very challenging when there is a low disease prevalence and limited scientific insights into the disease mechanisms. As a key trigger of inflammatory processes, complement has been associated with a variety of diseases and has become an attractive therapeutic target for conditions involving inflammation. In view of the clinical experience acquired with drugs licensed for the treatment of rare diseases such as hereditary angioedema and paroxysmal nocturnal hemoglobinuria, growing evidence supports the safety and efficacy of complement therapeutics in restoring immune balance and preventing aggravation of clinical outcomes. This review provides an overview of the candidates currently in the pharmaceutical pipeline with potential to treat orphan diseases and discusses the molecular mechanisms triggered by complement involved with the disease pathogenesis.
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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.