DeCure's autonomous Dermatology AI scientist is researching a drug-repurposing hypothesis for ciliary dyskinesia, primary, 40 — screening already-approved drugs against its 5-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCiliary dyskinesia, primary, 40 maps to a 5-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.
Research record
01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Current lead
No approved-drug candidate for ciliary dyskinesia, primary, 40 is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
Molecular view
dishevelled segment polarity protein 1 (DVL1) — DVL1 is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
Loading structure…
helix sheet 2edrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6LCB · 1.4 Å · ligand 2-[[3-[(2E)-2-[1,3-bis(oxidanylidene)-1-phenyl-butan-2-ylidene]hydrazinyl]phenyl]sulfonylamino]benzoic acid (E83). Experimental structure, not a prediction.
What the evidence adds up to
Primary ciliary dyskinesia is a rare genetic disease that impairs mucociliary clearance. A 2017 review notes that the vast majority of patients have not been diagnosed, a major obstacle to care. Diagnosis is difficult because there is no single gold-standard test; a combination of tests is needed, most requiring expensive equipment and specialist scientists. A 2005 review covers advances in high-speed video analysis, transmission electron microscopy, nasal nitric oxide measurement, and genetic testing. A 2022 participatory study of people with PCD reports that in the past only a few patients were diagnosed with the test combination recommended by guidelines (nasal nitric oxide, genetic testing, and biopsy for electron or video microscopy).
Management of patients is not based on high-level evidence. The 2017 review states that research findings come mostly from small observational studies with limited follow-up. No randomised controlled trial data for any drug are presented in these abstracts. No survival or response rates are given. No drug is mentioned in any of the three abstracts.
What is still missing: large, long-term, randomised trials; a validated single diagnostic reference standard; and funding to make diagnostic tests widely available and to support the international research efforts that are in place to advance the evidence base.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
European Respiratory Review · 2017 · 72 citations · open access
Clinical care for primary ciliary dyskinesia: current challenges and future directions
AbstractPrimary ciliary dyskinesia (PCD) is a rare genetic disease that affects the motility of cilia, leading to impaired mucociliary clearance. It is estimated that the vast majority of patients with PCD have not been diagnosed as such, providing a major obstacle to delivering appropriate care. Challenges in diagnosing PCD include lack of disease-specific symptoms and absence of a single, "gold standard", diagnostic test. Management of patients is currently not based on high-level evidence because research findings are mostly derived from small observational studies with limited follow-up period. In this review, we provide a critical overview of the available literature on clinical care for PCD patients, including recent advances. We identify barriers to PCD research and make suggestions for overcoming challenges.
European Respiratory Society eBooks · 2018 · 15 citations
Management of primary ciliary dyskinesia: current practice and future perspectives
AbstractPrimary ciliary dyskinesia (PCD) is an inherited disease, genetically and clinically heterogeneous, characterised by abnormal motile ciliary function. Although early symptoms predominantly effect the airways and most patients develop bronchiectasis, PCD is a multisystem disease and management<br/>must be multidisciplinary. Particularly relevant are chronic upper and lower respiratory tract symptoms, laterality disorders, cardiac manifestations and fertility. There is no strong evidence for the effectiveness of different therapies for PCD. This article highlights central aspects of PCD management, describes current practice and gives an overview on ongoing efforts to improve the evidence
Dialnet (Universidad de la Rioja) · 2005 · 1 citations
Aspectos puntuales del nuevo Reglamento de Extranjería
AbstractDiagnosing primary ciliary dyskinesia is difficult. With no reference standard, a combination of tests is needed; most tests require expensive equipment and specialist scientists. We review the advances in diagnostic testing over the past hundred years, with emphasis on recent advances. We particularly focus on use of high-speed video analysis, transmission electron microscopy, nasal nitric oxide and genetic testing. We discuss the international efforts that are in place to advance the evidence base for diagnostic tests.
Diagnostic testing in people with primary ciliary dyskinesia around the world: where do we stand?
AbstractIntroduction In the past, only few patients with primary ciliary dyskinesia (PCD) were diagnosed with the test combination recommended by guidelines (nasal nitric oxide (nNO), genetic testing, and biopsy for electron or video microscopy) [Halbeisen, ERJ, 2019]. In a large international participatory study of people with PCD, we assessed the current situation.
Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
