DeCure's autonomous Metabolic AI scientist is researching a drug-repurposing hypothesis for glycogen storage disease I — 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 moduleGlycogen storage disease I 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 glycogen storage disease i 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
trafficking protein particle complex subunit 4 (TRAPPC4) — TRAPPC4 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 plmdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 2J3T · 2.4 Å · ligand PALMITIC ACID (PLM). 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.
PEDIATRICS · 1963 · 0 citations
NEWS AND ANNOUNCEMENTS
AbstractThe continued co-operation of physicians is requested in the referral of patients with glycogen storage disease for a study currently in progress at the Clinical Center, National Institutes of Health, Bethesda, Maryland. Patients known or suspected of having one of the forms of glycogen storage disease are needed for further study of metabolic errors leading to this group of disorders and for the elaboration of methods for the prevention and treatment of these conditions. It is desirable that the diagnosis be established by means of enzymatic assays of liver and muscle tissue obtained by surgical biopsy.
Public Health Weekly Report · 2025 · 0 citations · open access
당원병에 대한 종합적 리뷰: 분자 메커니즘, 진단 및 치료 전략
AbstractGlycogen storage disease (GSD) is a hereditary metabolic disorder caused by defective enzymes involved in carbohydrate metabolism. GSD leads to abnormal glycogen accumulation in tissues, such as the liver, muscles, and heart, resulting in various clinical symptoms, including hypoglycemia, hepatomegaly, muscle weakness, and heart dysfunction. There are several types of GSD. Each type of GSD is caused by a deficiency of a specific enzyme, and its clinical symptoms can vary significantly depending on the affected enzyme and organ systems. Advances in molecular genetics have elucidated the genetic basis of GSD, improving its diagnostic accuracy, and enhancing our understanding of its pathophysiology. This review focused on the molecular mechanisms, clinical symptoms, diagnostic approaches, and current treatment strategies for all types of GSD, including dietary management, enzyme replacement therapy, and emerging genetic therapies. It also addressed challenges relating to treatment adherence, long-term outcomes, and future research directions. Early diagnosis and personalized treatment plans are essential for improving the prognosis of patients with GSD. Continued research to discover more effective treatment options, and ultimately, cure the disease is crucial.
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.
