Rare & Orphan Lab · DeCure for X

DeCure for Intellectual disability, autosomal dominant 6

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for intellectual disability, autosomal dominant 6 — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module1 genesLead labRare & Orphan
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Rare & OrphanDOID:0070036$DeCureRare

The disease map

Disease moduleIntellectual disability, autosomal dominant 6 maps to a 1-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 intellectual disability, autosomal dominant 6 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

glutamate ionotropic receptor NMDA type subunit 2B (GRIN2B)GRIN2B 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 glydrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 9OOS · 3.03 Å · ligand GLYCINE (GLY). Experimental structure, not a prediction.

What the evidence adds up to

The four abstracts provided do not address autosomal dominant intellectual disability type 6 specifically. One 2011 review discusses the Down syndrome behavioural phenotype, noting relative strengths in visual-spatial skills and relative challenges in motor planning, but offers no drug data. A second 2011 review on Fragile X syndrome states that intellectual disability has been considered immutable and that current medical practices aim only at relieving symptoms, not altering cognitive deficits. That review mentions the possibility that pharmaceutical therapies targeting Fragile X syndrome might become a benchmark for CNS drug discovery, but provides no results from any trial.

A 2025 case report describes a 15-year-old boy with intellectual disability who displayed inappropriate and challenging sexual behaviour and was treated with GNRH analogues. The report states that traditional treatments often fail for such behaviour and that alternative options need exploration, but it gives no quantitative outcomes—no response rates, no survival data, no sample size beyond the single patient. A 2022 review of mouse models of Bardet-Biedl Syndrome notes that pharmacological interventions for intellectual disability are limited, partly due to poor understanding and heterogeneity, and that few mouse models exist.

No abstract reports any drug tested in patients with autosomal dominant intellectual disability type 6. No efficacy, no response rate, no survival benefit can be claimed for any compound in this condition. What is missing is any clinical trial designed for this specific genetic subtype, any funding directed at it, and any patient stratification that would allow a targeted drug to be tested.

Evidence

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

Occupational Therapy In Health Care · 2011 · 81 citations

The Down Syndrome Behavioral Phenotype: Implications for Practice and Research in Occupational Therapy

AbstractABSTRACT Down syndrome (DS) is the most common chromosomal cause of intellectual disability. The genetic causes of DS are associated with characteristic outcomes, such as relative strengths in visual-spatial skills and relative challenges in motor planning. This profile of outcomes, called the DS behavioral phenotype, may be a critical tool for intervention planning and research in this population. In this article, aspects of the DS behavioral phenotype potentially relevant to occupational therapy practice are reviewed. Implications and challenges for etiology-informed research and practice are discussed.

https://doi.org/10.3109/07380577.2010.535601
ACS Chemical Neuroscience · 2011 · 19 citations · open access

Fragile X Syndrome: An Update on Developing Treatment Modalities

AbstractIntellectual disability (ID; mental retardation) is considered an immutable condition. Current medical practices are aimed at relieving symptoms and not at altering the underlying cognitive deficits. Scientific advancements from the past decade have led to the exciting possibility that ID may now be treatable. Moreover, pharmaceutical therapies targeting the most common form of inherited ID, Fragile X syndrome (FXS), may become the new benchmark for central nervous system (CNS) drug discovery: seeking cures for neurodevelopmental disorders.

https://doi.org/10.1021/cn200019z
Psychopharmacology Bulletin · 2025 · 2 citations · open access

Case Report: A Case of Intellectual Disability with Inappropriate and Challenging Sexual Behavior that was Treated with GNRH Analogues

AbstractIntellectual Disability starts within the course of developmental stages and covers both intellectual and adaptive deficiencies in conceptual, social and applied fields. Individuals with intellectual disability experience many difficulties in social life due to challenging and inappropriate sexual behaviour. Suchdifficulties need to be addressed, reduced or treated. Traditional treatments often fail to treat and improve suchbehavior. Alternative treatment options need to be explored with studies conducted in this field. With this paper, we aimed to show and touch on alternative treatments for challenging and inappropriate behaviors of a 15-year old boy with intellectual disability, who was treated with GNRH analogues.

https://doi.org/10.64719/pb.4607
Iowa Research Online (The University of Iowa) · 2022 · 0 citations · open access

Behavioral and brain phenotypes of mouse models of Bardet-Biedl Syndrome

AbstractIntellectual disability (ID) is one of the most common neurodevelopmental disorders, affecting 1% of the population globally. Clinically, ID is characterized by a deficit in intellectual functioning and adaptive functioning. There are limited pharmacological interventions for ID, partially due to a poor understanding of ID and the heterogeneous nature of ID In addition, there are limited mouse models of ID.

https://doi.org/10.25820/etd.006578

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.