Dravet Syndrome UK
Cycle 3
Dravet Syndrome is a rare, life-long and life limiting neurological condition. It includes seizures, intellectual disability and a range of associated health conditions known as ‘comorbidities’.
Last updated 04/30/2026
Clinical
Disease Class
Channelopathies
Neurological diseases
Body Systems
Cardiovascular / Circulatory
Hematopoietic / Lymphatic / Immune
Muscular / Skeletal
Nervous / Sensory
Respiratory
Organs
Brain
Known Genetic Link
Yes, one or more genes directly cause the condition
Causative Genes
GABRA1
GABRG2
SCN1A
SCN1B
Contributory Genes
None specified / unknown
Type of Inheritance
De novo
Newborn Screening
No
Disease Mechanism(s)
Ion channel dysfunction
Age of Onset
Early childhood (age 1+-5)
Infancy (age 0-1)
Average Age at Diagnosis
Early childhood (age 1+-5)
Life Expectancy
Adulthood (age 18-64)
Affected Sex(es)
Female
Male
National Prevalence
1001-10000
Global Prevalence
Unknown
National Incidence
Less than 10
Global Incidence
Unknown
Symptoms / Phenotypes
abnormal brain features
abnormal pyramidal sign
attention disorders / ADHD
behavioral changes
developmental delay
feeding difficulties
gait abnormalities / gait disturbance
hyperactivity
intellectual disability
seizures / epilepsy
sleep disorders
speech delay
Biomarkers
None
Existing Therapies
Off-Label Drug Use
Regulatory Agency-Approved for Symptom Relief
· Antiseizure treatments: sodium valproate, clobazam, stiripentol, Cannabidiol, fenfluramine. Also Topiramate, Levetiracetam. Sodium channel blockers should not be used in DS.
Therapies in Development
Antisense oligonucleotide (ASO) therapy
Gene therapy
· 1 in clinical trial: ETX-101
Small molecule therapy (novel small molecule drugs)
· 1 in clinical trial: Bexicaserin. 2 approved: Cannabidiol, Stiripentol
Therapeutic Development Stages
Approved/Available
In clinical trials (Phase I, II, III, or IV)
In preclinical development
In research/exploratory phase
Therapeutic Development Role
Focus group participation or coordination
Meetings with regulators (e.g., FDA listening sessions, PFDD meetings)
Study material design and/or review (not protocol) — includes patient-facing materials such as informed consent
Study protocol design and/or review (includes selection of outcome measures)
Organizational & Research
Cell Lines
None
Cell Lines, share
N/A
Disease Model
None
Disease Model, share
N/A
Organizational Challenges
Not applicable
Clinical Trial Role
Focus group
Meeting with regulators
Study material design, review (not protocol)
Study protocol design, review
Clinical Trial Types
Phase 1
Phase 2
Phase 3
Biobank, Institution
None
Center of Excellence, Institution
None
Registry
No, we do not have a registry and have no plans to create one
Natural History Study
Yes, we have collaborated on a natural history study
Data Collected, Natural History Study
Clinical endpoints (outcomes)
Genetic data
Medication usage
Patient-reported data
Prospective data
Retrospective data
Platform, Natural History Study
Other
FDA Patient Listening Session
No
FDA Patient-Focused Drug Development (PFDD) Program
No
ICD Codes
Yes, we have an ICD-10 code specific to our exact disease
Yes, we have an ICD-11 code specific to our exact disease
Diagnostic Guidelines
Yes, we have guidance available on our website
Science Advisory Board Policies
Does not have an SAB
Research Network Policies
Has CRN and willing to share policies
Patient Priority Survey
No
Research Roadmap
We don't have a Research Roadmap
International Chapters
None
International Partners
Europe
North America
Other International Research Initiatives
Europe