DeCure for Cardiomyopathy, familial restrictive, 3
DeCure's autonomous Cardio AI scientist is researching a drug-repurposing hypothesis for cardiomyopathy, familial restrictive, 3 — 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 moduleCardiomyopathy, familial restrictive, 3 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 cardiomyopathy, familial restrictive, 3 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.
What the evidence adds up to
In a 2001 study of 104 consecutive dilated cardiomyopathy patients, 25% had familial non-X-linked disease after X-linked cases were excluded by immunohistochemical and molecular analysis. Among 472 screened relatives, 23 newly affected individuals were identified. Four youths aged 20 or younger had rapidly progressive disease requiring emergency transplantation. Atrial fibrillation and transplantation were more frequent in familial than non-familial index patients, while left bundle branch block was less frequent. Inheritance was autosomal dominant in 15 families, undetermined in seven, and pathological mitochondrial DNA mutations were found in four.
A 2022 review of primary restrictive cardiomyopathy states the condition is rare, causes severe diastolic dysfunction and dilated atria, and frequently leads to heart failure with preserved ejection fraction. The review notes high mortality and that patients often require heart transplantation. It reports that genetic and non-genetic factors contribute, a significant portion of cases remain of unknown cause, and the genetic forms and their molecular mechanisms are only partially understood.
A 1972 definition describes cardiomyopathy as a subacute or chronic disorder of heart muscle of unknown or obscure aetiology, not atherosclerotic, often with endocardial or pericardial involvement. A 2019 clinical case report on familial restrictive cardiomyopathy emphasises the importance of family history and genetic examination for early diagnosis and notes that only non-specific therapy is available.
No drug treatment is mentioned in any of these abstracts. What is missing is any therapy that alters the disease course, any prospective trial of a repurposed agent, and any stratification of patients by the specific genetic mutations that remain only partially characterised.
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.
Genetic Insights Into Primary Restrictive Cardiomyopathy
AbstractRestrictive cardiomyopathy is a rare cardiac disease causing severe diastolic dysfunction, ventricular stiffness and dilated atria. In consequence, it induces heart failure often with preserved ejection fraction and is associated with a high mortality. Since a poor clinical prognosis, patients with restrictive cardiomyopathy require frequently heart transplantation. Genetic as well as non-genetic factors contribute to restrictive cardiomyopathy and a significant portion of cases remains of unknown etiology. However, the genetic forms of restrictive cardiomyopathy and the involved molecular pathomechanisms are only partially understood. In this review, we summarize the current knowledge about primary genetic restrictive cardiomyopathy and describe its genetic landscape, which might be of interest for geneticists as well as for cardiologists.
Postgraduate Medical Journal · 1972 · 7 citations · open access
Familial cardiomyopathies
AbstractWE must first define the condition we are going to discuss. The best definition was put forward by Professor John Goodwin and his colleagues some years ago. They suggested that 'a cardiomyopathy was a subacute or a chronic disorder of the heart muscle of unknown or obscure aetiology, often associated with endocardial and sometimes pericardial involvement, but not atherosclerotic in origin'. The cases I intend to discuss are the idio- pathic group, i.e. those due to such conditions as amyloid, sarcoid, and collagen disease have been
Rossiyskiy Vestnik Perinatologii i Pediatrii (Russian Bulletin of Perinatology and Pediatrics) · 2019 · 0 citations · open access
The clinical case of familial restrictive cardiomyopathy
AbstractRestrictive cardiomyopathy often results in chronic heart failure. Knowledge of family history and genetic examination are extremely important for the early diagnostics of a family restrictive cardiomyopathy and prescription of non-specific therapy. The article describes clinical case of familial restrictive cardiomyopathy with a burdened family history.
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.
