Rare & Orphan Lab · DeCure for X

DeCure for Hearing loss, autosomal dominant 83

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hearing loss, autosomal dominant 83 — 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 module1 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:0070609$DeCureRare

The disease map

Disease moduleHearing loss, autosomal dominant 83 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 hearing loss, autosomal dominant 83 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.

What the evidence adds up to

No drug was studied in any of the provided abstracts. The 2012 prevalence study of a UK NHS clinic found that 6.7% of the clinical population and 0.7% of the general population had hearing loss greater than 70 dB averaged over 0.5, 1, and 2 kHz, based on 2,199 cases identified from a database of 32,781. The 2023 study on subclinical hearing loss reported that normal-hearing adults showed significantly worse tonal thresholds at 12 and 16 kHz and worse performance on digit triplet tests in noise compared to young subjects, with lower wave I and V amplitudes and higher wave V latencies on auditory brainstem response. A 2004 clinical genetic study of 144 patients with nonsyndromic hearing loss described the distribution of sex, type, degree, symmetry, laterality, progression, and inheritance pattern but gave no treatment outcomes.

The 2025 review of gene therapy for hereditary hearing loss discusses three strategies—gene replacement, gene suppression, and gene editing—and notes that clinical trial results for gene therapy in hereditary hearing loss have recently been approved, but it provides no concrete numbers on survival, response rates, or sample sizes from those trials. Another 2025 collection of 31 papers on hearing loss includes reviews of basic mechanisms, gene therapy, and clinical studies but again offers no quantitative efficacy data. A 1991 paper in Spanish confirms the complex genetic landscape of hearing loss and states that whole-exome sequencing cannot achieve a 100% diagnostic rate due to genetic heterogeneity.

What is still missing is any completed clinical trial that reports a measurable improvement in hearing thresholds, speech perception, or quality of life for any drug or gene therapy in autosomal dominant hearing loss 83. No randomised controlled trial, no patient stratification by genetic subtype, and no funding commitment for such a trial is described in these abstracts.

Evidence

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

International Journal of Audiology · 2012 · 32 citations

Prevalence & characteristics of severe and profound hearing loss in adults in a UK National Health Service clinic

AbstractLaura Turton*ab & Pauline Smithcda Hearing Link, Eastbourne, UKb The Centre for Hearing & Balance Disorders, University Hospitals Coventry and Warwickshire NHS Trust, Coventry, UKc Hearing Services Department, University Hospitals of Leicester NHS Trust, Leicester, UKd Medical Research Council Hearing and Communication Group, Royal Free London NHS Foundation Trust, London, UKCorrespondence: Laura Turton, Hearing Link, 27–28, The Waterfront, Eastbourne, East Sussex, BN23 5UZ, UK. E-mail:[email protected]: To estimate the prevalence of severe and profound hearing loss in a clinical population and to report the audiological and hearing-aid characteristics for this group, as well as outcome measures from use of hearing aids. Design: A retrospective observational study initially, followed by a postal Glasgow health status inventory (GHSI) to establish the patients functional outcomes. Study sample: A clinical database of 32 781 cases was interrogated from which 2199 cases of severe /profound hearing loss were identified. From these, an adult sample stratified in terms of age and gender of n = 302 was contacted. Results: An estimated 6.7% of the local clinical population and 0.7% of the general population were found to have hearing > 70 dB averaged over 0.5, 1, and 2 kHz. Most patients were fitted with bilateral hearing aids, using a non-linear prescription, and as a group they reported a high level of social support. Conclusions: This study has estimated the prevalence of severe and profound hearing loss as 6.7% of the clinical population, and 0.7% of the general population. This is consistent with previous work, although it probably underestimates the prevalence. Further work is indicated to strengthen the estimate.

https://doi.org/10.3109/14992027.2012.735376
Sensory Neuroscience · 2025 · 7 citations · open access

Update on Gene Therapy in the Treatment of Hereditary Hearing Loss

AbstractABSTRACT Gene therapy is a promising therapeutic approach for genetic disorders, involving genetic modification to repair or reconstruct faulty genetic material. It is particularly relevant to hereditary hearing loss (HHL), a common monogenic condition that can lead to congenital deafness. The recent approval of clinical trial results using gene therapy for HHL underscores the growing interest in this field. To further advance inner ear gene therapy and its application in genetic diseases, it is crucial to review the progress of gene therapy for HHL. This review focuses on the three major gene therapy strategies—gene replacement, gene suppression, and gene editing—highlighting their application across different monogenic disorders and successful preclinical trials in HHL. We summarize the primary gene therapy strategies used in recent years, discuss recent achievements in preclinical studies, and explore potential advancements in this field.

https://doi.org/10.1002/sen2.70004
Journal of Otology · 2023 · 5 citations · open access

Subclinical hearing loss associated with aging

AbstractObjective: Contribute to clarifying the existence of subclinical hearing deficits associated with aging. Design: In this work, we study and compare the auditory perceptual and electrophysiological performance of normal-hearing young and adult subjects (tonal audiometry, high-frequency tone threshold, a triplet of digits in noise, and click-evoked auditory brainstem response). Study sample: ." Results: presented significantly worse tonal thresholds in the high frequencies (12 and 16 kHz) and worse performance in the digit triplet tests in noise. In the electrophysiological test using the auditory brainstem response technique, the adult group presented significantly lower I and V wave amplitudes and higher V wave latencies at the supra-threshold level. At the threshold level, we observed a significantly higher latency in wave V in the adult group. In addition, in the partial correlation analysis, controlling for the hearing level, we observed a relationship (negative) between age and speech in noise performance and high-frequency thresholds. No significant association was observed between age and the auditory brainstem response. Conclusion: The results are compatible with subclinical hearing loss associated with aging.

https://doi.org/10.1016/j.joto.2023.05.002
American Journal of Audiology · 2004 · 1 citations

Clinical Genetic Study of 144 Patients With Nonsyndromic Hearing Loss

AbstractHearing loss constitutes an important category of congenital defects that can be isolated or part of the phenotypic spectrum of several syndromes. A clinical genetic study was performed on a sample of 144 patients with nonsyndromic hearing loss, establishing the sex distribution, type, degree, symmetry, laterality, progression, etiology, and, when possible, inheritance pattern.

https://doi.org/10.1044/1059-0889(2004/013)
Advanced Science · 2025 · 1 citations · open access

Hearing Loss: From Basic to Clinical Science

AbstractHearing loss (HL) affects over 1.5 billion people globally, with genetic factors accounting for ≈50% of congenital cases. Therefore, HL has become a global health issue, driving extensive research from basic science to clinical applications. This Special Collection includes a total of 31 papers, among which 9 are review papers, 21 are research article papers, 1 is a perspective paper, that highlight the basic mechanisms and possible protection methods of HL, the application of gene therapy for treating HL, and the clinical study and application in HL.

https://doi.org/10.1002/advs.202521526
Revista de occidente · 1991 · 0 citations

Proclamas de la antelación de la muerte

AbstractOur findings confirm the complex genetic landscape of hearing loss and the limitations of WES in achieving a 100% diagnostic rate, especially in conditions characterized by genetic heterogeneity. These results contribute to our understanding of the genetic basis of hearing loss and emphasize the need for further research and comprehensive genetic analyses to elucidate the underlying causes of this condition.

https://doi.org/10.1186/s40246-024-00630-8

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.