Rare & Orphan Lab · DeCure for X

DeCure for Autosomal dominant nonsyndromic hearing loss 1

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for autosomal dominant nonsyndromic hearing loss 1 — 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:0110541$DeCureRare

The disease map

Disease moduleAutosomal dominant nonsyndromic hearing loss 1 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 autosomal dominant nonsyndromic hearing loss 1 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

The abstracts provided do not describe any drug treatment or clinical trial for autosomal dominant nonsyndromic hearing loss. The 1996 study of a large North American family documents a mutation causing progressive sensorineural hearing loss beginning around age 20 and becoming profound by about age 45, based on evaluation of 49 family members. The 2004 study of 144 patients with nonsyndromic hearing loss describes the clinical characteristics of the condition—sex distribution, type, degree, symmetry, laterality, progression, and inheritance pattern—but reports no intervention. A 2020 review notes that 119 nonsyndromic genes have been associated with hearing loss and that gene therapy is emerging as a possible future management option, but no specific drug or therapy is tested or recommended.

No evidence of any drug being repurposed, tested, or shown to alter hearing loss in this condition appears in these abstracts. The 2000 review states that hearing loss in older adults is usually multifactorial, while monogenic forms can be autosomal dominant, but again no treatment data are presented. The abstracts contain no response rates, survival figures, or efficacy claims because no drug was administered to any patient in these studies.

What is still missing is any clinical trial testing a drug for this specific mutation, any funding for such a trial, and any patient stratification that would identify which genetic subtype might respond to a given compound. The abstracts only describe natural history and genetic cataloguing, not therapeutic intervention.

Evidence

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

New England Journal of Medicine · 2000 · 202 citations

Genetic Causes of Hearing Loss

AbstractHearing loss is the most common sensory defect in humans, affecting normal communication in 10 percent of people aged 65 years or older. In most cases, hearing loss is a multifactorial disorder caused by both genetic and environmental factors. However, single-gene mutations can lead to hearing loss. In these cases, hearing loss is a monogenic disorder with an autosomal dominant, autosomal recessive, X-linked, or mitochondrial mode of inheritance. These monogenic forms of hearing loss can be syndromic (characterized by hearing loss in combination with other abnormalities) or nonsyndromic (with only hearing loss). This review focuses on nonsyndromic hearing loss, since . . .

https://doi.org/10.1056/nejm200004133421506
American Journal of Audiology · 1996 · 24 citations

Autosomal-Dominant Progressive Sensorineural Hearing Loss in a Large North American Family

AbstractForty-nine members of a family with autosomal-dominant progressive sensorineural hearing loss were evaluated by audiologists, otologists, and geneticists. The results presented here show a nonsyndromic, autosomal-dominant mutation causing progressive sensorineural hearing loss beginning at about age 20 and becoming profound by approximately age 45. Because of the unambiguous nature of the hearing loss, the size of the family, and the availability of two previously described temporal bones from family members, a fairly complete description of the nature and impact of this mutation will be presented.

https://doi.org/10.1044/1059-0889.0501.105
Laryngoscope Investigative Otolaryngology · 2020 · 7 citations · open access

Genetics of pediatric hearing loss: A functional perspective

AbstractOBJECTIVES: This article reviews the current role of genetics in pediatric hearing loss (HL). METHODS: A review of the current literature regarding the genetic basis of HL in children was performed. RESULTS: To date, 119 nonsyndromic genes have been associated with HL. There are also hundreds of syndromic causes that have HL as part of the clinical phenotype. CONCLUSIONS: Identifying HL genes coupled with clinical characteristics ("genotype-phenotype") yields a more accurate diagnosis and prognosis. Although the complexity of the auditory apparatus presents challenges, gene therapy is emerging and may be a viable management option in the future.

https://doi.org/10.1002/lio2.390
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)

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.