Disease Directory KCNQ2-Related Epilepsy
Neurological

KCNQ2-Related Epilepsy

Also known as: KCNQ2 channelopathy, KCNQ2 epileptic encephalopathy, benign neonatal epilepsy (mild variant)

Prevalence

1-9 per 100,000 (Orphanet)

Onset

Neonatal

Type

Autosomal dominant genetic (de novo in most severe cases)

Gene

KCNQ2

About KCNQ2-Related Epilepsy

KCNQ2-related epilepsy encompasses a spectrum from benign familial neonatal epilepsy to severe KCNQ2 epileptic encephalopathy, caused by mutations in KCNQ2 encoding the Kv7.2 potassium channel subunit. The severe encephalopathic form (de novo mutations) presents in the first days of life with tonic seizures, EEG burst-suppression pattern, and later neurodevelopmental impairment. Ezogabine (retigabine) directly opens Kv7.2 channels; sodium channel blockers and carbamazepine are often effective.

Common Clinical Features

Neonatal tonic seizures Burst-suppression EEG pattern Hypotonia Developmental delay and intellectual disability Movement disorder Visual abnormalities Absent speech

Clinical Trial Eligibility Tips

What to know before applying to KCNQ2-Related Epilepsy trials.

KCNQ2 pathogenic variant class (gain-of-function versus loss-of-function) determines treatment approach and trial eligibility for targeted therapies

EEG documentation of neonatal seizures and burst-suppression pattern is required for severe encephalopathy trials

Carbamazepine or phenobarbital response in the neonatal period is important history — document efficacy and tolerability

Gene therapy and channel opener (Kv7.2 activator) trials require no prior investigational KCNQ2-targeted treatment

Patient Resources

Patient Organization

KCNQ2 Cure Alliance

Visit website ↗

Natural History Registry

KCNQ2 Cure Alliance Patient Registry

Join registry ↗

Orphanet

European reference resource for rare diseases (ORPHA:439218)

View on Orphanet ↗

NORD

National Organization for Rare Disorders

Search NORD ↗

Find recruiting KCNQ2-Related Epilepsy trials

Search 500,000+ studies from ClinicalTrials.gov, filtered for KCNQ2-Related Epilepsy. Updated daily.

Related Rare Diseases