Rare & Orphan Lab · DeCure for X

DeCure for Multiple system atrophy

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for multiple system atrophy — screening already-approved drugs against its 27-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module27 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:4752$DeCureRare

The disease map

Disease moduleMultiple system atrophy maps to a 27-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 multiple system atrophy 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

myeloperoxidase (MPO)MPO 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 hemdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 5MFA · 1.2 Å · ligand PROTOPORPHYRIN IX CONTAINING FE (HEM). Experimental structure, not a prediction.

What the evidence adds up to

Multiple system atrophy is a rare, sporadic, adult-onset, progressive and fatal neurodegenerative disorder characterised by autonomic and motor dysfunction. The 2022 International Parkinson and Movement Disorder Society criteria enable an accurate and early diagnosis of clinically established MSA, clinically probable MSA, prodromal possible MSA, and definite pathologic diagnosis. A 2023 practical guide explains that these criteria include new categories for clinically established MSA and possible prodromal MSA, which may reduce time to diagnosis. The clinical diagnosis is based on symptoms of autonomic dysfunction with levodopa-resistant parkinsonism or cerebellar ataxia, alongside neuroimaging characteristics and exclusion of mimics. A 2010 article from Siberian State Medical University presents clinical and diagnostic criteria and a clinical case, but provides no quantitative results.

A 2017 case study of a 54-year-old woman with MSA presenting diplopia, gait disturbance, ataxia, sleep apnea, and dysuria reported notable improvement in diplopia expressing time and Unified Multiple System Atrophy Rating Scale (UMSARS) after treatment with Korean medical treatment including herbal medicine, acupuncture, bee venom acupuncture, electroacupuncture, cupping, and moxa. This is a single case with no control, and the authors state only that Korean medical treatment may be effective. No other treatment studies with quantitative outcomes are reported in these abstracts.

Management of MSA remains symptomatic, focusing on control of parkinsonism, ataxia, autonomic dysfunction, and other motor and nonmotor symptoms, with an updated multidisciplinary and multisystem approach including palliative care. The 2025 review notes that advances in brain imaging and molecular biomarker research and efforts to develop disease-modifying agents have shown promise, but no disease-modifying treatment is described. What is still missing are completed trials of disease-modifying agents, validated biomarkers for early diagnosis and monitoring, and patient stratification methods to identify who might benefit from specific symptomatic or experimental therapies.

Evidence

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

Practical Neurology · 2023 · 68 citations · open access

Multiple system atrophy

AbstractThis is a practical guide to diagnosing and managing multiple system atrophy (MSA). We explain the newly published Movement Disorders Society Consensus Diagnostic Criteria, which include new 'Clinically Established MSA' and 'Possible Prodromal MSA' categories, hopefully reducing time to diagnosis. We then highlight the key clinical features of MSA to aid diagnosis. We include a list of MSA mimics with suggested methods of differentiation from MSA. Lastly, we discuss practical symptom management in people living with MSA, including balancing side effects, with the ultimate aim of improving quality of life.

https://doi.org/10.1136/pn-2020-002797
The Journal of Internal Korean Medicine · 2017 · 3 citations · open access

A Case Study of Multiple System Atrophy Patient with Diplopia Using Korean Medical Treatment

AbstractIn this study, a 54-year-old female woman diagnosed as Multiple System Atrophy (MSA) presented diplopia with other symptoms -gait disturbance, ataxia, sleep apnea, dysuria. She had been cared with Korean medical treatment -Herbal medicine, acupuncture, bee venom acupuncture, electroacupuncture, cupping, moxa. Notable improvement was observed in diplopia expressing time and Unified Multiple System Atrophy Rating Scale (UMSARS). For MSA patient with no typical treatment indispensable, Korean medical treatment may be effective.

https://doi.org/10.22246/jikm.2017.38.2.246
CONTINUUM Lifelong Learning in Neurology · 2025 · 1 citations

Multiple System Atrophy

AbstractOBJECTIVE: This article provides up-to-date diagnosis and management concepts for patients with multiple system atrophy, a rare, sporadic, adult-onset, progressive, and fatal neurodegenerative disorder that is characterized mainly by autonomic and motor dysfunction. LATEST DEVELOPMENTS: Making an accurate and early diagnosis of multiple system atrophy remains challenging because of its clinical complexity and similarity in presentation to other neurodegenerative diseases. The clinical diagnosis of multiple system atrophy is based on the patient's symptoms of autonomic dysfunction with levodopa-resistant parkinsonism or cerebellar ataxia, alongside neuroimaging characteristics and exclusion of mimics. The 2022 International Parkinson and Movement Disorder Society criteria enable an accurate and early diagnosis of clinically established multiple system atrophy, clinically probable multiple system atrophy, prodromal possible multiple system atrophy, and the definite pathologic diagnosis of multiple system atrophy. The management of multiple system atrophy remains symptomatic in the control of parkinsonism, ataxia, autonomic dysfunction, and other motor and nonmotor symptoms, with an updated multidisciplinary and multisystem approach including palliative care. Advances in brain imaging and molecular biomarker research and efforts to develop disease-modifying agents have shown promise to improve diagnosis and treatment of this disorder. ESSENTIAL POINTS: Updated standards guide the clinical diagnosis and management of multiple system atrophy with a multidisciplinary and multisystem approach, and this article summarizes clinical best practices and emerging advances in multiple system atrophy.

https://doi.org/10.1212/cont.0000000000001598
Bulletin of Siberian Medicine · 2010 · 0 citations · open access

Multisystem atrophy: clinical manifestations, pathogenesis questions

AbstractThe article examines the pathogenesis and clinical features of multisystem atrophy. We present clinical and diagnostic criteria of the disease. The clinical signs of multisystem atrophy patients treated at the Neurological clinic Siberian State Medical University (Tomsk). The clinical case was examined.

https://doi.org/10.20538/1682-0363-2010-4-100-106
Oxford University Press eBooks · 2017 · 0 citations

Multiple System Atrophy

AbstractMultiple system atrophy is a group of diseases characterized by parkinsonism, ataxia, and dysautonomia. Although the etiology remains unclear, the molecular pathology has been somewhat clarified. The diagnosis is largely clinical but certain tests may help with confirming diagnosis. Treatment largely remains symptomatic.

https://doi.org/10.1093/med/9780199937837.003.0004

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.