Rare & Orphan Lab · DeCure for X

DeCure for Thrombocytopenia-absent radius syndrome

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for thrombocytopenia-absent radius 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 module2 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:14699$DeCureRare

The disease map

Disease moduleThrombocytopenia-absent radius 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 thrombocytopenia-absent radius 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.

Evidence

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

Special Care in Dentistry · 2013 · 3 citations

Thrombocytopenia absent radius (TAR) syndrome: a case report and review for oral health care providers

AbstractThrombocytopenia Absent Radius (TAR) syndrome is a rare genetic disorder characterized by thrombocytopenia and bilateral absence of radii with both thumbs present. Multiple organ systems may be affected in patients with TAR syndrome, including hematologic, skeletal, cardiovascular, and gastrointestinal systems. This article describes a 35-year-old Caucasian male with TAR syndrome receiving comprehensive dental care and reviews the pertinent aspects of this disease, as well as dental management considerations, for patients with TAR syndrome.

https://doi.org/10.1111/scd.12060
The Journal of Pediatric Research · 2017 · 0 citations · open access

Late Preterm Infant Diagnosed with Thrombocytopenia Absent Radius Syndrome

AbstractThrombocytopenia absent radius (TAR) syndrome is a rare genetic anomaly presenting with bilateral radial aplasia and thrombocytopenia. Pathophysiology is not fully understood. Patients may need to receive thrombocyte transfusions for better neurological outcome and frequent follow-up may prevent serious complications such as intracranial hemorrhage. Here we report a one-day-old late preterm neonate diagnosed with TAR syndrome by clinical and laboratory data, and confirmed by genetic studies.

https://doi.org/10.4274/jpr.57070

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.