Rare & Orphan Lab · DeCure for X

DeCure for Double outlet right ventricle

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for double outlet right ventricle — screening already-approved drugs against its 4-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module4 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:6406$DeCureRare

The disease map

Disease moduleDouble outlet right ventricle maps to a 4-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 double outlet right ventricle 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

MYCN proto-oncogene, bHLH transcription factor (MYCN)MYCN 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 adpdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 5G1X · 1.72 Å · ligand ADENOSINE-5'-DIPHOSPHATE (ADP). Experimental structure, not a prediction.

What the evidence adds up to

The four abstracts cover right ventricular function, right heart dysfunction, the embryological origins of double outlet right ventricle, and a separate rare condition called double chambered left ventricle. None of them test or even mention any drug for double outlet right ventricle. The 2012 embryological study of 35 hearts with double outlet right ventricle describes the anatomical variants as arising from progressive detorsion of the truncoconal septum, with measured twists of 135°, 90°, 0°, and −90° depending on the great artery arrangement. No treatment, drug, or intervention is evaluated in that paper. The 2022 report on double chambered left ventricle describes two cases, one congenital and one acquired, both managed surgically with an uneventful peri-operative period, but this is a different disease. The 2020 and 2018 reviews discuss the role of the right ventricle in cardiac performance and therapeutic strategies for right ventricular dysfunction in general, but they do not address double outlet right ventricle specifically and provide no drug data for that condition.

No evidence of any drug being studied for double outlet right ventricle appears in these abstracts. There are no response rates, survival figures, or sample sizes for any pharmacological intervention. The only management described is surgical, and that is for a different entity. The embryological work offers a classification of anatomical variants but no therapeutic direction.

What is missing is any clinical trial, any drug repurposing study, any animal model testing a compound, and any patient stratification strategy for double outlet right ventricle. Funding for preclinical drug screening or for a registry that could link specific anatomical subtypes to outcomes is absent from this literature. Without such data, no pharmacological claim can be made.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

American Journal of Physiology-Heart and Circulatory Physiology · 2020 · 16 citations · open access

Synergy in the heart: RV systolic function plays a key role in optimizing LV performance during exercise

AbstractThe right ventricle appears to have an important impact on maintaining systemic cardiac function and delivering stroke volume. However, its exact role in supporting left ventricular function has so far been unclear. This study demonstrates a new mechanism of ventricular interaction that provides mechanistic understanding of the key importance of the right ventricle in driving cardiac performance.

https://doi.org/10.1152/ajpheart.00256.2020
Journal of Cardiovascular Medicine · 2018 · 11 citations

Right heart dysfunction: from pathophysiologic insights to therapeutic options: a translational overview

Abstract: The right ventricle has become increasingly studied in cardiovascular research. In this article, we describe specific pathophysiological characteristics of the right ventricle, with special focus on functional and molecular modifications as well as therapeutic strategies in right ventricular dysfunction, underlining the differences with the left ventricle. Then we analyze the main imaging modalities to assess right ventricular function in different clinical settings. Finally, we acknowledge main therapeutic advances for treatment of right heart diseases.

https://doi.org/10.2459/jcm.0000000000000700
Archivos de cardiología de México · 2012 · 4 citations · open access

Doble salida de ventrículo derecho. Enfoque embriológico

AbstractOBJECTIVE: It is proposed a pathogenetic explanation that explains the morphogenesis of the anatomic variants of double outlet right ventricle. METHOD: An anatomic embryological correlation was made in which the plane separating the outlets and great arteries in the types of this cardiopathy was compared with the normal truncoconal septum in the embryonic heart. Thirty five hearts with double outlet right ventricle were described, fifteen with great arteries slightly crossed, ten with side by side great arteries and ten with anterior aorta and posterior pulmonary artery. The cephalic border of the truncoconal septum was compared with its inferior border in each group. With this procedure we calculated the type of torsion of the truncoconal septum. RESULTS: In the slightly crossed great arteries the truncoconal twist was of 135° in side by side great arteries the twist was of 90° and in anterior right aorta the truncoconal septum was straight with 0° of rotation, and with left anterior aorta the rotation was of -90°. CONCLUSION: Embryologically double outlet right ventricle is originated by the persisting continuity between the right ventricle with the truncus and conus which form the great arteries and their outlets. The anatomic variations are the consequence of progressive detortion of the truncoconal septum followed by a torsion of -90°.

https://doi.org/10.1016/j.acmx.2012.09.002
Journal of Cardiac Surgery · 2022 · 3 citations

Double chambered left ventricle—institutional experience of two cases

AbstractBACKGROUND: Double Chambered Left Ventricle is a very rare congenital cardiac condition with limited cases reported in literature, etiology of which is obscure and presentation can be varied. AIM OF THE STUDY: To share our experience of two diverse cases of Double Chambered Left Ventricle at our institute. RESULTS: Two diverse cases of Double Chambered Left Ventricle were appropriately diagnosed and successfully managed. One of which one was congenital and the other was acquired METHODS: Both the patients had an uneventful peri-operative period. CONCLUSIONS: Double Chambered Left Ventricle is a condition which remains an enigma. Diagnosis requires a high degree of radiological suspicion for prompt diagnosis and good surgical outcomes.

https://doi.org/10.1111/jocs.16966

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.