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Disease · MONDO_0010790

MERRF syndrome

6 sourcesdiseases-2026-09-18-0.1.0

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ConductScience Library. MERRF syndrome. Diseases, record MONDO_0010790. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010790

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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
MERRF syndrome
Open Targets
Open Targets ID
MONDO_0010790
Open Targets
Other names
Fukuhara syndrome, MERRF, myoclonic epilepsy - ragged red fibres, myoclonic epilepsy- ragged red fibers, myoclonus epilepsy and ragged red fibres, myoclonus epilepsy associated with ragged-red fibers, myoclonus epilepsy associated with ragged-red fibres, myoclonus with epilepsy and with ragged Red fibers (MERRF syndrome), myoclonus with epilepsy and with ragged Red fibres, myoclonus with epilepsy and with ragged Red fibres (MERRF syndrome)
Open Targets
Disease Ontology
DOID:310
Disease Ontology
MeSH
D017243
MeSH
NCI Thesaurus
C84889
NCI Thesaurus
Orphanet
ORPHA:551
Orphanet
Highest stage of its drugs
Phase 2, for its one drug, from ClinicalTrials.gov
Open Targets

Drugs1

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.

DrugChEMBL IDTypeOpen Targets' stageStage reported byReportsOf them trialsTrials shown here
CYSTEAMINE BITARTRATECHEMBL1200577Small moleculePhase 2ClinicalTrials.gov111

Targets0

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.

None in this release.

Trials1

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.

TrialTitlePhaseParticipantsResults first postedConditionsDrugsLinked by
NCT02473445A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial DiseasePhase 2222018-05-11Mitochondrial DiseasesCYSTEAMINE BITARTRATEOpen Targets' reading of its record

Comparisons in its trials0

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.

None in this release.

Papers from its trials0

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

None in this release.

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_0010790

GET https://conductscience.org/api/diseases/v1/MONDO_0010790/drugs