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fragile X syndrome
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Disease · MONDO_0010383

Targets103

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.

1 to 20 of 103

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TargetProteinIts drugs that act on itMechanismsHighest stage of those drugsGene in GenesSource
GABBR1GABA-B receptor3GABA-B receptor agonistPhase 3https://conductscience.org/library/genes/2550Drugs and targets
GABBR2GABA-B receptor3GABA-B receptor agonistPhase 3https://conductscience.org/library/genes/9568Drugs and targets
GABRA2GABA-A receptor; anion channel3GABA receptor alpha-2 subunit positive allosteric modulator; GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2555Drugs and targets
GABRA3GABA-A receptor; anion channel3GABA receptor alpha-3 subunit positive allosteric modulator; GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2556Drugs and targets
GABRA4GABA-A receptor; anion channel3GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator; Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonistPhase 2https://conductscience.org/library/genes/2557Drugs and targets
GABRB3GABA-A receptor; anion channel3GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator; Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonistPhase 2https://conductscience.org/library/genes/2562Drugs and targets
GABRDGABA-A receptor; anion channel3GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator; Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonistPhase 2https://conductscience.org/library/genes/2563Drugs and targets
GRM5Metabotropic glutamate receptor 53Metabotropic glutamate receptor 5 antagonist; Metabotropic glutamate receptor 5 modulator; Metabotropic glutamate receptor 5 negative allosteric modulatorPhase 2/3https://conductscience.org/library/genes/2915Drugs and targets
DRD2D(2) dopamine receptor2Dopamine D2 receptor partial agonist; Dopamine receptor agonistPhase 2https://conductscience.org/library/genes/1813Drugs and targets
GABRA1GABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2554Drugs and targets
GABRA5GABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2558Drugs and targets
GABRA6GABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2559Drugs and targets
GABRB1GABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2560Drugs and targets
GABRB2GABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2561Drugs and targets
GABREGABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2564Drugs and targets
GABRG1GABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2565Drugs and targets
GABRG2GABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2566Drugs and targets
GABRG3GABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2567Drugs and targets
GABRPGABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/2568Drugs and targets
GABRQGABA-A receptor; anion channel2GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulatorPhase 2https://conductscience.org/library/genes/55879Drugs and targets

diseases-2026-09-18-0.1.0

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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.

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