Disease · MONDO_0007113
Angelman syndrome
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. Angelman syndrome. Diseases, record MONDO_0007113. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0007113
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.
- Highest stage of its drugs
- Phase 3, for 3 of its 15 drugs, from ClinicalTrials.gov and TTD
- Open Targets
Drugs15
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 |
|---|---|---|---|---|---|---|---|---|
| RUGONERSEN | CHEMBL5095469 | Oligonucleotide | Phase 3 | ClinicalTrials.gov | 5 | 3 | 3 | Drugs and targets |
| GABOXADOL | CHEMBL312443 | Small molecule | Phase 3 | ClinicalTrials.gov and TTD | 4 | 3 | 3 | Drugs and targets |
| OBUDANERSEN SODIUM | CHEMBL6068612 | Oligonucleotide | Phase 3 | ClinicalTrials.gov | 4 | 2 | 2 | Drugs and targets |
| LEVODOPA | CHEMBL1009 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| MINOCYCLINE | CHEMBL1434 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| ALOGABAT | CHEMBL5095259 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| NNZ-2591 | CHEMBL4802144 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| CARBIDOPA | CHEMBL1200748 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| BETAINE | CHEMBL95889 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| CREATINE | CHEMBL283800 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 15
See all 15 drugsTargets7
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 |
|---|---|---|---|---|---|---|
| BHMT | Betaine-homocysteine S-methyltransferase | 1 | Betaine-homocysteine S-methyltransferase substrate | Unknown | https://conductscience.org/library/genes/635 | Drugs and targets |
| BHMT2 | Betaine-homocysteine S-methyltransferase | 1 | Betaine-homocysteine S-methyltransferase substrate | Unknown | https://conductscience.org/library/genes/23743 | Drugs and targets |
| DDC | Aromatic-L-amino-acid decarboxylase | 1 | DOPA decarboxylase inhibitor | Phase 1 | https://conductscience.org/library/genes/1644 | Drugs and targets |
| DRD3 | D(3) dopamine receptor | 1 | Dopamine D3 receptor agonist | Phase 2/3 | https://conductscience.org/library/genes/1814 | Drugs and targets |
| GABRA4 | Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta | 1 | Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonist | Phase 3 | https://conductscience.org/library/genes/2557 | Drugs and targets |
| GABRB3 | Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta | 1 | Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonist | Phase 3 | https://conductscience.org/library/genes/2562 | Drugs and targets |
| GABRD | Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta | 1 | Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonist | Phase 3 | https://conductscience.org/library/genes/2563 | Drugs and targets |
Trials16
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 |
|---|---|---|---|---|---|---|---|---|
| NCT03882918 | An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome | Phase 3 | 141 | 2025-07-18 | Angelman Syndrome | GABOXADOL | Its record names the disease | ClinicalTrials.gov |
| NCT04106557 | A Study of OV101 in Individuals With Angelman Syndrome (AS) | Phase 3 | 104 | 2024-01-08 | Primary Disease or Condition Being Studied: Angelman Syndrome (AS) | GABOXADOL | Its record names the disease | ClinicalTrials.gov |
| NCT00348933 | Dietary Supplements for the Treatment of Angelman Syndrome | Not applicable | 90 | 2012-09-24 | Angelman Syndrome, Nervous System Diseases | BETAINE, CREATINE, LEVOMEFOLATE CALCIUM, LEVOMEFOLIC ACID, VITAMIN B12 | Its record names the disease | ClinicalTrials.gov |
| NCT02996305 | A Study in Adults and Adolescents With Angelman Syndrome (STARS) | Phase 2 | 88 | 2022-03-31 | Angelman Syndrome | GABOXADOL | Its record names the disease | ClinicalTrials.gov |
| NCT01281475 | A Trial of Levodopa in Angelman Syndrome | Phase 2/3 | 67 | 2017-01-13 | Angelman Syndrome | LEVODOPA | Its record names the disease | ClinicalTrials.gov |
| NCT01531582 | Minocycline in the Treatment of Angelman Syndrome | Not applicable | 25 | 2023-04-18 | Angelman Syndrome | MINOCYCLINE | Its record names the disease | ClinicalTrials.gov |
| NCT00829439 | Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome | Phase 1 | 16 | 2016-11-21 | Angelman Syndrome | CARBIDOPA, LEVODOPA | Its record names the disease | ClinicalTrials.gov |
| NCT07605429 | BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. | Phase 3 | 165 | Angelman Syndrome | RUGONERSEN | Its record names the disease | ClinicalTrials.gov | |
| NCT06914609 | REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome | Phase 3 | 156 | Angelman Syndrome | OBUDANERSEN SODIUM | Its record names the disease | ClinicalTrials.gov | |
| NCT04428281 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS) | Phase 1 | 74 | Angelman Syndrome | RUGONERSEN | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 16
See all 16 trialsComparisons 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 |
|---|---|---|---|---|---|
| Levodopa vs Placebo: serious adverse events | 3 of 33 | 1 of 34 | 67 | NCT01281475 | Trial comparisons |
| Levodopa vs Placebo: Presence of Tremors | 14 of 29 | 12 of 26 | 55 | NCT01281475 | Trial comparisons |
Papers from its trials8
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_0007113
GET https://conductscience.org/api/diseases/v1/MONDO_0007113/drugs