DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for movement disorder — screening already-approved drugs against its 37-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMovement disorder maps to a 37-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 movement disorder 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
protein tyrosine phosphatase receptor type D (PTPRD) — PTPRD 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 flcdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 2YD6 · 1.35 Å · ligand CITRATE ANION (FLC). Experimental structure, not a prediction.
What the evidence adds up to
Of 51 children seen in a single paediatric genetic movement disorder clinic, 26 (51%) received a confirmed genetic diagnosis. Among those, 20 had ataxia and 6 had dystonia. Targeted next-generation sequencing panels identified a genetic diagnosis in 9 patients, and whole exome sequencing identified one in 14 patients. In 10 of the 26 patients with a genetic diagnosis, the finding led to either disease-specific treatment or a change in management.
The clinical spectrum of paediatric movement disorders is broad and heterogeneous, ranging from acute self-limited conditions to conditions causing lifelong motor disability. Most childhood movement disorders are chronic, and the large number of rare genetic conditions poses a significant diagnostic challenge. Recognition of distinctive diagnostic clues in history and examination can facilitate diagnosis of potentially treatable disorders.
Movement disorders encompass a wide variety of diagnoses and clinical situations. Most patients are managed in the outpatient setting, but certain conditions present urgently or emergently, where missed or delayed diagnosis can have severe consequences. These movement disorder emergencies form a distinct category requiring prompt recognition.
What is still missing is a prospective trial design that tests whether early genetic diagnosis and targeted management actually improves long-term motor outcomes in paediatric movement disorders, rather than simply altering management in the short term. The necessary funding for such a trial, and a reliable method for stratifying patients by genetic subtype before treatment assignment, have not been established.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Neurology Genetics · 2018 · 40 citations · open access
Genetic landscape of pediatric movement disorders and management implications
AbstractOBJECTIVE: To identify underlying genetic causes in patients with pediatric movement disorders by genetic investigations. METHODS: All patients with a movement disorder seen in a single Pediatric Genetic Movement Disorder Clinic were included in this retrospective cohort study. We reviewed electronic patient charts for clinical, neuroimaging, biochemical, and molecular genetic features. DNA samples were used for targeted direct sequencing, targeted next-generation sequencing, or whole exome sequencing. RESULTS: There were 51 patients in the Pediatric Genetic Movement Disorder Clinic. Twenty-five patients had dystonia, 27 patients had ataxia, 7 patients had chorea-athetosis, 8 patients had tremor, and 7 patients had hyperkinetic movements. A genetic diagnosis was confirmed in 26 patients, including in 20 patients with ataxia and 6 patients with dystonia. Targeted next-generation sequencing panels confirmed a genetic diagnosis in 9 patients, and whole exome sequencing identified a genetic diagnosis in 14 patients. CONCLUSIONS: We report a genetic diagnosis in 26 (51%) patients with pediatric movement disorders seen in a single Pediatric Genetic Movement Disorder Clinic. A genetic diagnosis provided either disease-specific treatment or effected management in 10 patients with a genetic diagnosis, highlighting the importance of early and specific diagnosis.
CONTINUUM Lifelong Learning in Neurology · 2019 · 16 citations
Movement Disorders in Children
AbstractPURPOSE OF REVIEW: This article provides an overview of the clinical features and disorders associated with movement disorders in childhood. This article discusses movement disorder phenomena and their clinical presentation in infants and children and presents a diagnostic approach to suspected genetic disorders with a focus on treatable conditions. RECENT FINDINGS: Technologic advances in molecular genetic testing over the past decade continue to lead to the discovery of new diseases. This article discusses the clinical presentation and early experience with treatment for several recently described genetic forms of infantile-onset and childhood-onset dystonia and chorea. SUMMARY: The clinical spectrum of pediatric movement disorders is broad and heterogeneous, ranging from acute or transient self-limited conditions to conditions that cause profound lifelong motor disability. Most movement disorders in childhood are chronic, and the large number of rare, genetic conditions associated with pediatric movement disorders can pose a significant diagnostic challenge. Recognition of distinctive diagnostic clues in the history and examination can facilitate the diagnosis of potentially treatable disorders.
AbstractMovement disorders encompass a wide variety of diagnoses and clinical situations. Most patients with movement disorders are managed in the outpatient setting, and usually, time is not of the essence. However, certain conditions present urgently or emergently, and the consequences of missed or delayed diagnosis can be severe. We call these conditions movement disorder emergencies, and their diagnosis and management form the topic of this book.
BMJ Case Reports · 2021 · 2 citations · open access
Bilateral chorea following severe traumatic brain injury treated with risperidone
AbstractA functionally independent man in his 20s with a history of intellectual disability and epilepsy and family history of Huntington's disease suffered a severe traumatic brain injury. Postinjury, bilateral chorea rendered him dependent for all activities of daily living. Risperidone provided a significant reduction of chorea, decreasing the overall burden of care. Movement disorders are a common sequela of brain injury. Currently, there are no best treatment guidelines for chorea in patients with brain injury. To the authors' knowledge there have been no case reports describing the effects of brain injury on patients with a primary movement disorder. Risperidone was an effective treatment in this case. Further research is needed to establish guidelines for treatment of movement disorders following brain injury and to better understand the effect of brain injuries on primary movement disorders.
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.
