Disease · MONDO_0003608

optic atrophy

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. optic atrophy. Diseases, record MONDO_0003608. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0003608

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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.

Name: optic atrophy; Open Targets
Open Targets ID: MONDO_0003608; Open Targets
Other names: Optic nerve atrophy, Optic-nerve degeneration, atrophy of optic disk; Open Targets
Disease Ontology: DOID:5723; Disease Ontology
MeSH: D009896; MeSH
NCI Thesaurus: C34863; NCI Thesaurus
Highest stage of its drugs: Phase 2, for 1 of its 5 drugs, from ClinicalTrials.gov; 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
METFORMIN	CHEMBL1431	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
DARBEPOETIN ALFA	CHEMBL1201566	Protein	Phase 1/2	ClinicalTrials.gov	1	1	0	Drugs and targets
EPOETIN ALFA	CHEMBL1201565	Protein	Phase 1/2	ClinicalTrials.gov	1	1	0	Drugs and targets
EPOETIN BETA	CHEMBL2109092	Protein	Phase 1/2	ClinicalTrials.gov	1	1	0	Drugs and targets

Targets54

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
EPOR	Erythropoietin receptor	3	Erythropoietin receptor agonist	Phase 1/2	https://conductscience.org/library/genes/2057	Drugs and targets
COXFA4	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/4697	Drugs and targets
COXFA4L2	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/56901	Drugs and targets
GPD2	Glycerol-3-phosphate dehydrogenase, mitochondrial	1	Mitochondrial glycerol-3-phosphate dehydrogenase inhibitor	Phase 2	https://conductscience.org/library/genes/2820	Drugs and targets
MT-ND1	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/4535	Drugs and targets
MT-ND2	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/4536	Drugs and targets
MT-ND3	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/4537	Drugs and targets
MT-ND4	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/4538	Drugs and targets
MT-ND4L	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/4539	Drugs and targets
MT-ND5	Mitochondrial complex I (NADH dehydrogenase)	1	Mitochondrial complex I (NADH dehydrogenase) inhibitor	Phase 2	https://conductscience.org/library/genes/4540	Drugs and targets

1 to 10 of 54

See all 54 targets

Trials2

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
NCT05426044	Metformin as a Neuroprotective Therapy for Glaucoma	Phase 2	125		Glaucoma	METFORMIN	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 trials1

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it
34185708	RESULT	1

Source PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

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_0003608

GET https://conductscience.org/api/diseases/v1/MONDO_0003608/targets?page=2

API reference For AI assistants