DeCure for Cardiomyopathy, familial restrictive, 6
DeCure's autonomous Cardio AI scientist is researching a drug-repurposing hypothesis for cardiomyopathy, familial restrictive, 6 — 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 moduleCardiomyopathy, familial restrictive, 6 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 cardiomyopathy, familial restrictive, 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
kinesin family member 20A (KIF20A) — KIF20A 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 ipadrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6YIP · 1.43 Å · ligand ISOPROPYL ALCOHOL (IPA). Experimental structure, not a prediction.
What the evidence adds up to
Of 104 consecutive patients with dilated cardiomyopathy, 25% had familial disease after excluding X-linked cases. Among 472 screened relatives, 23 newly affected individuals were identified. Familial cases more often required transplantation (p=0.04) and had atrial fibrillation (p=0.04) than non-familial cases. Inheritance was autosomal dominant in 15 families, undetermined in seven, and linked to mitochondrial DNA mutations in four. Four youths aged 20 or younger had rapidly progressive disease requiring emergency transplantation.
Restrictive cardiomyopathy in childhood is rare, defined by diastolic dysfunction without systolic impairment or ventricular dilatation. A single case report describes a 15-year-old presenting with heart failure signs. The broader WHO classification distinguishes dilated, hypertrophic, restrictive, and arrhythmogenic right ventricular cardiomyopathies. Dilated cardiomyopathy remains the most common cause of chronic heart failure, but its exact incidence, time course, and pathogenetic mechanisms are unknown; viral and autoimmune involvement are recognised possible causes.
No abstract reports any drug treatment or intervention for familial restrictive cardiomyopathy. No trial data, response rates, or survival figures exist in these abstracts for any pharmacological therapy. The 2001 screening study did not test any drug; it only diagnosed asymptomatic relatives.
What is missing: any randomised trial of a drug for familial restrictive cardiomyopathy, any preclinical model testing a repurposed compound, any biomarker to stratify patients by genetic subtype, and any funding for such a trial. Without these, the evidence base remains diagnostic only.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
European Heart Journal · 2001 · 31 citations · open access
Evidence-based diagnosis of familial non-X-linked dilated cardiomyopathy. Prevalence, inheritance and characteristics
AbstractAIMS: To assess the prevalence of familial non-X-linked dilated cardiomyopathy, to diagnose early asymptomatic cases evaluate inheritance and characterize clinical phenotypes. METHODS AND RESULTS: We screened 472 relatives of 104 consecutive patients diagnosed with dilated cardiomyopathy; males with X-linked dilated cardiomyopathy were excluded based on systematic immunohistochemical and molecular analysis. Relatives underwent clinical examination, electrocardiography, echocardiography and serum creatine-phosphokinase determination. Twenty-six index patients (25%) had familial disease: four youths (< or = 20 years) had rapidly progressive outcome and underwent emergency transplantation. In a sib-pair, the onset was with atrioventricular block. Inheritance was autosomal dominant in 15, undetermined in seven (four sib-pairs); mitochondrial DNA pathological mutations were found in four. The screening identified 23 newly diagnosed relatives in the familial group. Transplantation (P = 0.04) and atrial fibrillation (P = 0.04) were more frequent, and left bundle branch block (P = 0.04) less frequent in index patients with familial than in those with non-familial disease. Several non-affected relatives had instrumental abnormalities potentially useful as pre-clinical markers: their prevalence was similar in both groups. CONCLUSIONS: The prevalence of familial, non X-linked dilated cardiomyopathy was 25%. The immediate benefits of screening family members of index patients was early diagnosis in unaware symptomless affected relatives.
Mechanical Circulatory Support · 1997 · 2 citations
What is dilated cardiomyopathy?
AbstractCardiomyopathies are diseases of the myocardium with cardiac dysfunction not due to specific causes such as hypertension, congenital, valvular, coronary, arterial, or pericardial abnormalities. According to the definition of the WHO, primary cardiomyopathies are classified as dilated cardiomyopathy, hypertrophic cardiomyopathy, restrictive cardiomyopathy, and arrhythmogenic right ventricular cardiomyopathy (18). Dilated cardiomyopathy is the most common cause of chronic heart failure. The involved pathogenetic mechanisms, the incidence and exact time course of the disease are yet unknown. It may be idiopathic or familial/genetic although specific causes such as a viral and/or (auto)immune involvement of the heart have been recognized as a possible cause of the disease.
The Annals of Clinical and Analytical Medicine · 2013 · 0 citations · open access
Restrictive Cardiomyopathy in Childhood: Case Report
AbstractRestrictive cardiomyopathy is quite rare in childhood. The disease is characterized by diastolic dysfunction without systolic dysfunction or ventricular dilatation.Right heart failure signs, tromboembolism and cardiac arrhytmia can be seen in clinic presentation. In this case report, a 15 year-old patient who had heart failure signs was diagnosed with restrictive cardiomyopathy .
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.
