Disease · MONDO_0010382

fragile X-associated tremor/ataxia syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. fragile X-associated tremor/ataxia syndrome. Diseases, record MONDO_0010382. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010382

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.

Name: fragile X-associated tremor/ataxia syndrome; Open Targets
Open Targets ID: MONDO_0010382; Open Targets
Other names: FXTAS syndrome, Fragile X tremor/ataxia syndrome, X-linked dominant; Open Targets
Disease Ontology: DOID:0050879; Disease Ontology
MeSH: C564105; MeSH
NCI Thesaurus: C126566; NCI Thesaurus
Orphanet: ORPHA:93256; Orphanet
Highest stage of its drugs: Phase 2, for 2 of its 3 drugs, from ClinicalTrials.gov; Open Targets

Drugs3

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
BREXANOLONE	CHEMBL207538	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
CITICOLINE	CHEMBL1231700	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
MEMANTINE	CHEMBL807	Small molecule	Unknown	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets23

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
GABRA1	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2554	Drugs and targets
GABRA2	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2555	Drugs and targets
GABRA3	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2556	Drugs and targets
GABRA4	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2557	Drugs and targets
GABRA5	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2558	Drugs and targets
GABRA6	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2559	Drugs and targets
GABRB1	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2560	Drugs and targets
GABRB2	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2561	Drugs and targets
GABRB3	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2562	Drugs and targets
GABRD	GABA-A receptor; anion channel	1	GABA-A receptor; anion channel positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2563	Drugs and targets

1 to 10 of 23

See all 23 targets

Trials3

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.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00584948	Memantine Treatment in Fragile X-Associated Tremor/Ataxia Syndrome	Not applicable	94	2017-04-10	Fragile X-Associated Tremor/Ataxia Syndrome, Fragile X Premutation Carriers	MEMANTINE	Its record names the disease	ClinicalTrials.gov
NCT02197104	Citocoline for Treatment in Fragile X-associated Tremor/Ataxia Syndrome	Phase 2	10	2022-11-02	Fragile X Tremor/Ataxia Syndrome	CITICOLINE	Its record names the disease	ClinicalTrials.gov
NCT02603926	Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone	Phase 2	6	2018-12-06	Fragile X-associated Tremor/Ataxia Syndrome	BREXANOLONE	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials1

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
Memantine vs Placebo: serious adverse events	2 of 47	3 of 47	94	NCT00584948

Source Trial comparisons

Papers from its trials2

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	Source
32053612	DERIVED	1	PubMed
24345444	DERIVED	1	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_0010382

GET https://conductscience.org/api/diseases/v1/MONDO_0010382/targets?page=2

API reference For AI assistants