DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Riley-Day syndrome — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleRiley-Day syndrome maps to a 2-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 riley-day 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
serine palmitoyltransferase long chain base subunit 1 (SPTLC1) — SPTLC1 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 vsddrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7K0K · 2.6 Å · ligand 3-Dehydrosphinganine (VSD). 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.
Clinical Pediatrics · 1966 · 4 citations
Review : Familial Dysautonomia: The Riley-Day Syndrome
AbstractAlthough individual case reports had already appeared, it remained for Riley in 1949 to recognize the common clinical features of these and his own patients as a definite disease entity. This is a review of that disease—the Riley-Day syndrome, or familial dysau tonomia. This report includes a summary of the clinical pattern, laboratory aids to diagnosis, differential diagnosis, current therapy and prognosis.
Contemporary Perioperative Management of Adult Familial Dysautonomia (Riley-Day Syndrome)
AbstractFamilial dysautonomia (Riley-Day syndrome) is a rare multisystem disorder associated with an excess risk of perioperative morbidity and mortality. Because life expectancy is limited, few reports consider the perioperative management of familial dysautonomia in adults with advanced disease and end-organ dysfunction. Here, we report on the management of an adult patient with familial dysautonomia, highlighting recent developments in perioperative technology and pharmacology of special relevance to this challenging population.
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.
