Rare & Orphan Lab · DeCure for X

DeCure for Proximal myopathy with extrapyramidal signs

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for proximal myopathy with extrapyramidal signs — 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:0111335$DeCureRare

The disease map

Disease moduleProximal myopathy with extrapyramidal signs 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 proximal myopathy with extrapyramidal signs 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

mitochondrial calcium uptake 1 (MICU1)MICU1 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 cdldrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 6K7Y · 3.6 Å · ligand CARDIOLIPIN (CDL). 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.

Postgraduate Medical Journal · 1979 · 25 citations · open access

Epsilon aminocaproic acid (EACA) myopathy

AbstractTwo female patients developed a severe, painful proximal myopathy after taking 18--30 g of epsilon-aminocaproic acid daily for 5 weeks. Marked elevations of serum aminotransferases, creatine kinase and aldolase levels were found and the first patient had electromyographic and muscle biopsy changes of an acute monophasic, necrotising myopathy at the height of the illness. Resolution occurred in both cases on stopping the drug and the second patient had no electromyographic or muscle biopsy abnormalities 3 weeks later. Only 2 recognized cases of the condition have been reported previously but a review of the literature revealed several other possible examples.

https://doi.org/10.1136/pgmj.55.642.282
Zenodo (CERN European Organization for Nuclear Research) · 2023 · 0 citations · open access

Newly Diagnosed Patient with Anti-Hmgcoa Antibody- Associated Necrotizing Autoimmune Myopathy (Immune-Mediated Necrotizing Myopathy IMNM)

AbstractAnti- HMGR antibody positive immune mediated necrotizing myopathy is a rare disease was first recognized and characterised in patients with a history of stating exposure and immune -mediated necrotizing myopathy. We reported a rare case of patient presenting with features of proximal myopathy which has a wide differential diagnoses of which a diagnosis of anti- HMGCR-antibody associated myopathy should not be neglected. Effective treatment can Improve the prognosis if diagnosed in time, We provide a summary of clinical findings, pathological features, muscle imaging and immunogenetic risk factors of the disease. We also discuss treatment strategies and approaches to monitoring the therapeutic response. lastly, we briefly summaries the current understanding of the pathophysiology of the disease and postulate a mode for autoimmunity initiation and propagation in the disease.

https://doi.org/10.5281/zenodo.8053858

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.