DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for familial amyloid neuropathy — screening already-approved drugs against its 3-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleFamilial amyloid neuropathy maps to a 3-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 familial amyloid neuropathy 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
transthyretin (TTR) — TTR 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 2,4-dimethylphenyldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8C86 · 1.1 Å · ligand (2,4-dimethylphenyl)(4-hydroxy-3-methoxy-5-nitrophenyl)methanone (TQ0). 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.
Neurorehabilitation · 2021 · 0 citations · open access
Are pharmacological treatments for familial amyloid polyneuropathy effective and safe? A Cochrane Review summary with commentary
AbstractBACKGROUND: Familial amyloid polyneuropathies (FAPs) are a group of rare autosomal dominant transmitted disorders that can progressively lead to disability from neuropathy, autonomic failure and other system involvement. OBJECTIVE: The aim of this commentary is to discuss Cochrane evidence on the efficacy and safety of disease-modifying drugs (DMDs) for the treatment of FAPs from a rehabilitation perspective. METHODS: To summarize and discuss from a rehabilitation perspective the published Cochrane Review "Pharmacological treatment for familial amyloid polyneuropathy" by Magrinelli et al.RESULTS:This Cochrane review included 4 randomized controlled trials (RCTs) involving 655 adults with FAP. These four trials compared four different DMDs with placebo. The Cochrane Systematic Review reported that current evidence is limited. CONCLUSIONS: FAPs are a group of chronic disabling conditions in which a multidisciplinary approach, including an adequate rehabilitation programme along with a long-term effective pharmacological therapy, should always be envisaged.
Oxford University Press eBooks · 2014 · 0 citations
Familial amyloid polyneuropathy
AbstractThis chapter gives a thorough grounding in familial amyloid polyneuropathy. The term familial amyloid polyneuropathy refers to multisystem disorders transmitted as an autosomal dominant trait, involving nerve lesions induced by deposits of amyloid fibrils. Most cases are related to mutations of the transthyretin gene. Mutant apolipoprotein A 1 or gelsolin are rarer precursors of amyloidosis.
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.
