Rare & Orphan Lab · DeCure for X

DeCure for Moyamoya disease

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Moyamoya disease — screening already-approved drugs against its 34-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module34 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:13099$DeCureRare

The disease map

Disease moduleMoyamoya disease maps to a 34-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 moyamoya disease 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

RAP1A, member of RAS oncogene family (RAP1A)RAP1A 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 gtpdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 4KVG · 1.65 Å · ligand GUANOSINE-5'-TRIPHOSPHATE (GTP). Experimental structure, not a prediction.

What the evidence adds up to

Moyamoya disease has been recognised as a clinical entity for more than 40 years, but unresolved problems remain, including its true epidemiology, the genetic mechanism, and prevention of repeated haemorrhagic events. The disease is a chronic progressive cerebrovascular disorder defined by stenosis or occlusion of the bilateral internal carotid arteries, anterior cerebral arteries and middle cerebral arteries, accompanied by a collateral network of vessels at the base of the brain. Ischaemia and intracranial haemorrhage are the common typical manifestations, but atypical presentations such as headache, seizures and involuntary movements have also been reported.

A report from a tertiary care hospital in north-east India described six cases with varied presentations. Three patients presented with alternating hemiparesis, one had amaurosis fugax, one had ischaemic stroke with hemichorea, and one had focal seizure as the only manifestation. Two of these six cases notably had stenosis of the posterior cerebral artery in addition to the typical bilateral anterior circulation stenosis. The authors note that owing to its low incidence in India, moyamoya disease is easily overlooked as a possible diagnosis, and they emphasise the need for early diagnosis and surgical treatment.

An ongoing prospective multi-centre cohort study, the Asymptomatic Moyamoya Registry (AMORE) study, aims to clarify the prognosis of asymptomatic moyamoya disease and establish treatment strategies for these patients. A 2001 paper suggests that moyamoya disease may have a different presentation and a more benign natural history in the US population than in Asian populations, but this finding is based on limited data and the authors stress the need to better understand the natural history and the clinical benefit of different treatment modalities.

What is still missing are structured multi-centre randomised clinical trials to assess the best treatment modalities, particularly for patients outside Asia. The true epidemiology remains unclear, the genetic mechanism is not fully elucidated, and there is no proven strategy to prevent repeated haemorrhagic events. Patient stratification by asymptomatic versus symptomatic status, and by geographic or ethnic background, has not been systematically addressed in controlled trials.

Evidence

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

Neurologia medico-chirurgica · 2010 · 93 citations · open access

Moyamoya Disease: Recent Progress and Outlook

AbstractMoyamoya disease as a clinical entity has been known for more than 40 years. Constant efforts have been directed at clarifying the pathogenesis of this disorder and improving therapeutic methods for the ischemic and hemorrhagic stroke caused by the characteristic vasculopathy of this disease. Although much knowledge has been gained, unresolved problems remain, such as the true epidemiology of this disease, elucidation of the genetic mechanism, and prevention of repeated hemorrhagic events. In this paper, we review recent progress and discuss the outlook for this disorder.

https://doi.org/10.2176/nmc.50.824
Annals of Indian Academy of Neurology · 2014 · 9 citations · open access

Varied presentations of moyamoya disease in a tertiary care hospital of north-east India

AbstractINTRODUCTION: Moyamoya disease is a chronic progressive cerebrovascular disorder, characterized by stenosis or occlusion of bilateral internal carotid arteries (ICAs), anterior cerebral arteries (ACAs) and middle cerebral arteries (MCAs), accompanied by a collateral network of vessels formed at the base of the brain. Ischemia and intracranial hemorrhage are the common typical manifestations. However moyamoya disease has been associated with atypical presentations like headache, seizures and involuntary movements. Although frequently reported from Asian countries like Japan, China and Korea, only few studies reported on clinical manifestations of moyamoya disease from India. OBJECTIVES: To study the varied presentations of moyamoya disease in a tertiary care hospital of north-east India. MATERIAL AND METHODS: Relevant investigations were done to rule out other causes of moyamoya syndrome. RESULTS: We report 6 cases of moyamoya disease with varied presentations from a tertiary care referral government hospital. Case 1, 2 and 6 presented with alternating hemiparesis. Case 3 had amaurosis fugax. Case 4 had history suggestive of ischemic stroke and presented with hemichorea. Case 4 had focal seizure as the only manifestation. Cases 4 and 5 notably had stenosis of posterior cerebral artery (PCA) in addition to stenosis of bilateral ICAs, ACAs and MCAs. CONCLUSION: Owing to its low incidence in India, moyamoya disease is easily overlooked as a possible diagnosis. However, because of its progressive nature, it is imperative to diagnose this disease early and offer surgical treatment to the patients.

https://doi.org/10.4103/0972-2327.138518
Surgery for Cerebral Stroke · 2013 · 2 citations · open access

Concept of Prospective Multi-center Cohort Study on Prognosis of Asymptomatic Moyamoya Disease: Asymptomatic Moyamoya Registry (AMORE) Study

AbstractWe present the historical considerations on asymptomatic moyamoya disease and also introduce the concept of an on-going prospective multi-center cohort study on prognosis of asymptomatic moyamoya disease (Asymptomatic Moyamoya Registry; AMORE study). It is essential to clarify this issue and establish treatment strategies for patients with asymptomatic moyamoya disease in the very near future.

https://doi.org/10.2335/scs.41.235
Boletín del Instituto de Estudios Giennenses · 2001 · 0 citations

Fiestas de la ciudad de Jaén con motivo de la proclamación del Rey Fernando VI (1746)

AbstractMoyamoya disease may have a different presentation and more benign natural history in our population than in Asian populations. Our findings emphasize the need to better understand the natural history of patients with moyamoya as well as the clinical benefit of different treatment modalities. Structured multicenter randomized clinical trials are needed to further assess the best treatment modalities for patients with moyamoya in the United States.

https://doi.org/10.1001/archneur.58.8.1274

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.