Disease · MONDO_0018105
Wolfram syndrome
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. Wolfram syndrome. Diseases, record MONDO_0018105. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018105
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Other names
DIDMOAD, DIDMOAD syndrome, Diabetes insipidus - diabetes mellitus - optic atrophy - deafness, diabetes insipidus, diabetes mellitus, optic atrophy, and deafness syndrome, diabetes insipidus-diabetes mellitus-optic atrophy-deafness syndrome, diabetes mellitus and insipidus with optic atrophy and deafness
- Open Targets
Drugs5
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| SODIUM PHENYLBUTYRATE | CHEMBL1746 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| VALPROATE SODIUM | CHEMBL433 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| VALPROIC ACID | CHEMBL109 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| DANTROLENE SODIUM | CHEMBL928 | Small molecule | Phase 1/2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| EXENATIDE | CHEMBL414357 | Protein | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets4
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| ALDH5A1 | Succinate-semialdehyde dehydrogenase, mitochondrial | 2 | Succinate semialdehyde dehydrogenase inhibitor | Phase 2 | https://conductscience.org/library/genes/7915 | Drugs and targets |
| GLP1R | Glucagon-like peptide 1 receptor | 1 | Glucagon-like peptide 1 receptor agonist | Unknown | https://conductscience.org/library/genes/2740 | Drugs and targets |
| RYR1 | Ryanodine receptor 1 | 1 | Ryanodine receptor 1 antagonist | Phase 1/2 | https://conductscience.org/library/genes/6261 | Drugs and targets |
| RYR3 | Ryanodine receptor 3 | 1 | Ryanodine receptor 3 antagonist | Phase 1/2 | https://conductscience.org/library/genes/6263 | Drugs and targets |
Trials4
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 1 more is not shown, since the checks do not confirm it.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT02829268 | A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome | Phase 1/2 | 21 | 2024-03-13 | Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia | DANTROLENE SODIUM | Its record names the disease | ClinicalTrials.gov |
| NCT03717909 | Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome | Phase 2 | 63 | Wolfram Syndrome | VALPROIC ACID | Its record names the disease | ClinicalTrials.gov | |
| NCT04940572 | Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome | Phase 2 | 23 | Wolfram Syndrome | VALPROATE SODIUM | Its record names the disease | ClinicalTrials.gov | |
| NCT01302327 | GLP Analogs for Diabetes in Wolfram Syndrome Patients | Not applicable | 0 | Diabetes Mellitus Associated With Genetic Syndrome, Wolfram Syndrome | EXENATIDE | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials2
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Pediatric vs Adult: deaths from any cause | 0 of 8 | 0 of 11 | 19 | NCT02829268 | Trial comparisons |
| Pediatric vs Adult: serious adverse events | 0 of 8 | 0 of 11 | 19 | NCT02829268 | Trial comparisons |
Papers from its trials3
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0018105
GET https://conductscience.org/api/diseases/v1/MONDO_0018105/drugs