DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Dandy-Walker syndrome — screening already-approved drugs against its 15-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleDandy-Walker syndrome maps to a 15-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 dandy-walker syndrome 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
tubulin alpha 1a (TUBA1A) — TUBA1A 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 gtpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 9WD9 · 2.26 Å · ligand GUANOSINE-5'-TRIPHOSPHATE (GTP). Experimental structure, not a prediction.
What the evidence adds up to
Ten cases of Dandy-Walker syndrome are described in a 1990 report. Almost 80 percent of these children had associated anomalies. Satisfactory treatment in that series consisted mostly of shunting the lateral ventricular system to the peritoneum, but the incidence of complications was high. A separate 2000 series of 10 children found that a large posterior fossa on physical examination was the most reliable clinical finding, diagnostic in 9 out of 10 patients. The most satisfactory treatment in that series was a combination of a shunt from the lateral ventricle and the posterior fossa to the peritoneum.
A 2017 genetic analysis of two fetuses with Dandy-Walker malformation used single nucleotide polymorphism microarray (SNP-array). One fetus carried a 6p25.3p25.2 microdeletion, and another carried an Xp22.33p22.2 deletion and a Yq11.221q11 duplication. The abnormal fragments involved the FOXC1, SHOX, and STS genes, which the authors state are associated with Dandy-Walker malformation. The parents of both fetuses had a normal karyotype. The authors conclude that alteration of 6p25.3p25.2 and Xp22.33p22.2 copy numbers probably underlies the syndrome in these two fetuses, and that SNP-array can provide an important supplement for prenatal diagnosis.
No drug treatment is mentioned in any of these abstracts. The evidence consists entirely of small case series and two fetal genetic reports. There are no controlled trials, no survival statistics, and no response rates to any pharmacological intervention.
What is still missing is any trial of a drug therapy, any patient stratification beyond anatomical or genetic description, and any funding for a treatment study rather than further descriptive reports.
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 Journal of Pediatric Surgery · 1990 · 3 citations
The Dandy-Walker Syndrome
AbstractTen cases of the Dandy-Walker syndrome are presented. The clinical manifestations are analyzed. Almost 80 percent of these children had associated anomalies. The characteristic findings are based on the magnetic resonance image scan. Satisfactory treatment of our patients had mostly consisted in shunting the lateral ventricular system to the peritoneum. The incidence of the complications was high.
[Genetic analysis of two cases with Dandy-Walker deformed fetus].
AbstractOBJECTIVE: To explore the genetic etiology of two fetuses with Dandy-Walker malformation using single nucleotide polymorphism microarray (SNP-array). METHODS: The fetuses and their parents were subjected to G banding karyotype analysis. The fetuses were also subjected to SNP-array analysis. RESULTS: The parents of both fetuses showed a normal karyotype. One fetus has a 46,X,?i(X)(q10), while for another conventional cell culture has failed. SNP-array showed that one fetus carried a 6p25.3p25.2 microdeletion, and another carried a Xp22.33p22.2 deletion and a Yq11.221q11 duplication. The abnormal fragments have involved FOXC1, SHOX and STS genes, which are associated with Dandy-Walker malformation. CONCLUSION: Alteration of 6p25.3p25.2, Xp22.33p22.2 copy numbers probably underlies the Dandy-Walker syndrome in the fetuses. The disorder may be attributed to abnormal expression of FOXC1, SHOX, and STS genes. SNP-array can provide an important supplement for prenatal diagnosis.
FORMACION DE CAPA DE BASE RECICLADA CON EMULSION EN LA A-92
Abstract10 children with Dandy-Walker syndrome are presented to discuss their clinical assessment and therapy. The presence on physical examination of a large posterior fossa was the most reliable clinical finding and was diagnostic in 9 out of the 10 patients. The different diagnostic confirmatory studies are discussed. The most satisfactory treatment in this series was the combination of a shunt from the lateral ventricle and the posterior fossa to the peritoneum.
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.
