Disease · MONDO_0014960
encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy. Diseases, record MONDO_0014960. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0014960
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
- encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy
- Open Targets ID
- MONDO_0014960
- Other names
DS, Dravet syndrome, PEBEL, SMEI, Severe myoclonic epilepsy of infancy, Severe myoclonus epilepsy of infancy, encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy; PEBEL, encephalopathy, progressive, early-onset, with brain oedema and/or leukoencephalopathy; PEBEL
- Highest stage of its drugs
- Approval, for 3 of its 6 drugs, from DailyMed and FDA
Source Open Targets
Drugs6
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 |
|---|---|---|---|---|---|---|---|---|
| CANNABIDIOL | CHEMBL190461 | Small molecule | Approval | FDA | 8 | 7 | 7 | Drugs and targets |
| STIRIPENTOL | CHEMBL1983350 | Small molecule | Approval | DailyMed and FDA | 3 | 0 | 0 | Drugs and targets |
| FENFLURAMINE HYDROCHLORIDE | CHEMBL2106217 | Small molecule | Approval | DailyMed and FDA | 2 | 0 | 0 | Drugs and targets |
| ZOREVUNERSEN | CHEMBL5095386 | Oligonucleotide | Phase 2 | ClinicalTrials.gov | 3 | 2 | 2 | Drugs and targets |
| FENFLURAMINE | CHEMBL87493 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ZOREVUNERSEN SODIUM | CHEMBL5314626 | Oligonucleotide | Unknown | USAN | 1 | 0 | 0 | Drugs and targets |
Targets4
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 |
|---|---|---|---|---|---|---|
| HTR1D | 5-hydroxytryptamine receptor 1D | 2 | Serotonin 1d (5-HT1d) receptor agonist | Approval | https://conductscience.org/library/genes/3352 | Drugs and targets |
| HTR2C | 5-hydroxytryptamine receptor 2C | 2 | Serotonin 2c (5-HT2c) receptor agonist | Approval | https://conductscience.org/library/genes/3358 | Drugs and targets |
| SIGMAR1 | Sigma non-opioid intracellular receptor 1 | 2 | Sigma opioid receptor antagonist | Approval | https://conductscience.org/library/genes/10280 | Drugs and targets |
| CNR1 | Cannabinoid receptor 1 | 1 | Cannabinoid CB1 receptor negative allosteric modulator | Approval | https://conductscience.org/library/genes/1268 | Drugs and targets |
Trials9
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 |
|---|---|---|---|---|---|---|---|---|
| NCT02224573 | An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Adults With Dravet or Lennox-Gastaut Syndromes | Phase 3 | 681 | 2022-02-03 | Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome | CANNABIDIOL | Its record names the disease | ClinicalTrials.gov |
| NCT02224703 | GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome | Phase 3 | 199 | 2019-08-01 | Epilepsy, Dravet Syndrome | CANNABIDIOL | Its record names the disease | ClinicalTrials.gov |
| NCT02091375 | Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1) | Phase 3 | 120 | 2018-07-20 | Epilepsy, Dravet Syndrome | CANNABIDIOL | Its record names the disease | ClinicalTrials.gov |
| NCT02091206 | A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1) | Phase 2 | 34 | 2018-07-19 | Epilepsy, Dravet Syndrome | CANNABIDIOL | Its record names the disease | ClinicalTrials.gov |
| NCT04611438 | Research on Cognitive Effect of Cannabidiol on Dravet Syndrome and Lennox-Gastaut SyndromeGastaut Syndrome | Phase 3 | 107 | Dravet Syndrome, Lennox Gastaut Syndrome | CANNABIDIOL | Its record names the disease | ClinicalTrials.gov | |
| NCT04442295 | An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome | Phase 1/2 | 62 | Dravet Syndrome | ZOREVUNERSEN | Its record names the disease | ClinicalTrials.gov | |
| NCT04740476 | An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome | Phase 2 | 60 | Dravet Syndrome | ZOREVUNERSEN | Its record names the disease | ClinicalTrials.gov | |
| NCT03467113 | A Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With Dravet Syndrome or Lennox Gastaut Syndrome Currently Taking Cannabidiol | Phase 1 | 9 | Dravet Syndrome, Lennox Gastaut Syndrome | CANNABIDIOL, FENFLURAMINE | Its record names the disease | ClinicalTrials.gov | |
| NCT02318563 | Cannabidiol Oral Solution as an Adjunctive Therapy for Treatment of Participants With Inadequately Controlled Dravet Syndrome | Phase 3 | 0 | Dravet Syndrome | CANNABIDIOL | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials5
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 |
|---|---|---|---|---|---|
| Dravet Syndrome vs Lennox-Gastaut Syndrome: deaths from any cause | 6 of 315 | 12 of 366 | 681 | NCT02224573 | Trial comparisons |
| Dravet Syndrome vs Lennox-Gastaut Syndrome: serious adverse events | 133 of 315 | 157 of 366 | 681 | NCT02224573 | Trial comparisons |
| GWP42003-P 20 mg/kg/Day Dose vs Placebo: Number Of Participants With A ≥50% Reduction From Baseline In Convulsive Seizure Frequency During The Treatment Period | 26 of 61 | 16 of 59 | 120 | NCT02091375 | Trial comparisons |
| GWP42003-P 20 mg/kg/Day Dose vs Placebo: Number Of Participants Using Rescue Medication | 36 of 61 | 41 of 59 | 120 | NCT02091375 | Trial comparisons |
| GWP42003-P 20 mg/kg/Day Dose vs Placebo: serious adverse events | 10 of 61 | 3 of 59 | 120 | NCT02091375 | Trial comparisons |
Papers from its trials7
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_0014960
GET https://conductscience.org/api/diseases/v1/MONDO_0014960/drugs