DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Noonan syndrome 7 — 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 moduleNoonan syndrome 7 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 noonan syndrome 7 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
B-Raf proto-oncogene, serine/threonine kinase (BRAF) — BRAF 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 agsdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8VYP · 3.29 Å · ligand PHOSPHOTHIOPHOSPHORIC ACID-ADENYLATE ESTER (AGS). Experimental structure, not a prediction.
What the evidence adds up to
Noonan syndrome is a genetic disorder of autosomal dominance with an estimated prevalence of 1:1000 to 1:2500 live births. Typical features include short stature, cardiovascular abnormalities, characteristic facial deformity, learning difficulties, developmental delay, bleeding manifestations, and lymphatic malformation. Dental features reported include malocclusion, dental caries, giant cell and cystic lesions. A 2025 overview from Kyrgyzstan notes that data from Central Asia remain limited and emphasises the role of growth hormone therapy in treatment, while also highlighting the importance of early detection for improving prognosis. The same overview is published twice in the provided abstracts.
A 2018 report on auditory electrophysiological tests in a 5-year-old Malay boy with Noonan syndrome who could only produce a few meaningful words found bilateral mild conductive hearing loss at low frequencies. Auditory brainstem response recordings showed good waveform morphology but atypical results: absolute latency of wave V was normal, interpeak latencies of waves I-V, I-II, and II-III were prolonged, and interpeak latency of waves III-V was abnormally shorter. The authors note that 55 years after the syndrome was described, there was no publication regarding the use of auditory electrophysiological tests for analysing the central auditory nervous system in Noonan syndrome patients.
A 2015 case report of a 26-month-old boy diagnosed with Noonan syndrome with sporadic inheritance secondary to advanced paternal age states that multidisciplinary approach is the key to success in managing these children. A 2021 case report of a 6-year-old boy similarly states that multidisciplinary treatment plays a key role in overall quality of life. No clinical trial data, no drug efficacy data, and no survival or response rate numbers are provided in any of these abstracts. What is still missing are large-scale prospective studies, standardised auditory evaluation protocols, and any controlled trials of growth hormone or other interventions in this population.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Dental considerations and management in Noonan Syndrome: A case report with review of literature
AbstractNoonan syndrome is a genetic disorder of autosomal dominance with an estimated prevalence of 1:1000 – 1:2500 live birth. The typical features include short stature, cardiovascular abnormalities and characteristics facial deformity. Dental features reported so far include malocclusion, dental caries, giant cell and cystic lesion. Multidisciplinary treatment plays a key role in the overall quality of life of the patient. This case report describes a 6-year-old boy with Noonan syndrome.
Function and Disability Journal · 2018 · 0 citations · open access
The Results of ABR in a Child with Noonan Syndrome: Necessity of Renewing the Evaluation Protocols of Syndromes
Abstract55 years after discovering NS, there was not even one publication regarding the use of auditory electrophysiological tests for analyzing the central auditory nervous system in NS patients. This is an attempt to attract attention of scientists and clinicians in using AEPs for evaluating the function of CANS in NS. Readers can find a report about the results of audiological tests and auditory brainstem response (ABR) findings in a 5-year old Malay boy with NS. It should be noted that he could only produce a few meaningful words. The results of audiological tests showed bilateral mild conductive hearing loss at low frequencies. ABR recordings showed good waveform morphology but the results were atypical. That is, absolute latency of wave V was normal but interpeak latencies of waves I-V, I-II, II-III were prolonged. Conversely, interpeak latency of waves III-V was abnormally shorter.
Journal of Evolution of Medical and Dental Sciences · 2015 · 0 citations · open access
NOONAN SYNDROME: AN EARLY DIAGNOSIS
AbstractNoonan syndrome is a genetic multisystem disorder characterised by facial dysmorphism, learning difficulties, developmental delay, short stature, cardiac defects, bleeding manifestations and lymphatic malformation affecting 1 in 1000-2500 children. This is a case report of a twenty six month old boy diagnosed to have Noonan syndrome with sporadic inheritance secondary to advanced paternal age. Multidisciplinary approach is the key to success in managing these children, who are diagnosed at an early age.
Zenodo (CERN European Organization for Nuclear Research) · 2025 · 0 citations · open access
Синдром Нунан: клинический случай, значение диагностики в Кыргызстане
AbstractNoonan syndrome is a relatively common genetic disorder characterized by distinctive facial features, congenital heart defects, short stature, and developmental anomalies. Despite the growing number of reported cases worldwide, data from Central Asia remain limited. This article presents an overview of Noonan syndrome with a focus on current approaches to diagnosis and treatment, including the role of growth hormone therapy. We also provide insights into the occurrence of the syndrome in Kyrgyzstan, discuss possible complications, and highlight the importance of early detection for improving prognosis.
Zenodo (CERN European Organization for Nuclear Research) · 2025 · 0 citations · open access
Синдром Нунан: клинический случай, значение диагностики в Кыргызстане
AbstractNoonan syndrome is a relatively common genetic disorder characterized by distinctive facial features, congenital heart defects, short stature, and developmental anomalies. Despite the growing number of reported cases worldwide, data from Central Asia remain limited. This article presents an overview of Noonan syndrome with a focus on current approaches to diagnosis and treatment, including the role of growth hormone therapy. We also provide insights into the occurrence of the syndrome in Kyrgyzstan, discuss possible complications, and highlight the importance of early detection for improving prognosis.
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.