Rare & Orphan Lab · DeCure for X

DeCure for Bilateral perisylvian polymicrogyria

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for bilateral perisylvian polymicrogyria — screening already-approved drugs against its 7-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module7 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:0080924$DeCureRare

The disease map

Disease moduleBilateral perisylvian polymicrogyria maps to a 7-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 bilateral perisylvian polymicrogyria 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.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Neurology · 1999 · 59 citations

Bilateral perisylvian polymicrogyria in three generations

AbstractA family is described in which bilateral perisylvian polymicrogyria was present in 6 members of 3 consecutive generations. Typical anatomic and clinical findings of the syndrome, with a mild phenotype, were present in the 5 affected women from all 3 generations. More severe impairment was observed in the only affected male individual, a boy, in the third generation. Analysis of the pedigree and severity of the phenotype in the affected boy are consistent with transmission of an X-linked dominant trait, although other patterns of inheritance cannot be ruled out with certainty.

https://doi.org/10.1212/wnl.52.9.1910
Neuropediatrics · 2019 · 0 citations

MAST1-Gene Variations: A New Monogenetic Neuronal Migration Disorder Causing Congenital Bilateral Perisylvian Syndrome (CBPS)

AbstractBackground: Congenital bilateral perisylvian syndrome (CBPS) is a rare cortical migration disorder associated with typical clinical and imaging features such as bilateral symmetrical polymicrogyria, either exclusively or mainly affecting the perisylvian region of the brain. Clinical features of CBPS often include oromotor dysfunction, epilepsy and variable degrees of developmental delay. There are multiple non-genetic causes of polymicrogyria and CBPS in particular, including vascular and hypoxemic insults or congenital infections. Up to now genetic causes have been rarely reported, mostly associated with syndromic forms or peroxisomal disorders.

https://doi.org/10.1055/s-0039-1698269

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.