About Dravet Syndrome
Dravet syndrome is a severe developmental and epileptic encephalopathy caused in approximately 80% of cases by de novo loss-of-function mutations in SCN1A, encoding the Nav1.1 sodium channel subunit. Onset occurs in the first year of life with prolonged febrile and afebrile seizures, followed by developmental plateau and multiple seizure types that are refractory to conventional antiseizure medications. Sodium channel blockers (e.g., carbamazepine, phenytoin) worsen seizures and are contraindicated.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Dravet Syndrome trials.
SCN1A variant classification (pathogenic, likely pathogenic) and functional impact are required for gene therapy trial eligibility
Sodium channel blockers are contraindicated — ensure current antiseizure medication list is provided; trials screen for contraindicated drugs
Seizure frequency diary (minimum 3 months) is a standard inclusion criterion — document baseline convulsive seizure count
Fenfluramine (Fintepla) and cannabidiol (Epidiolex) are approved add-on therapies — prior treatment history affects trial stratification
Patient Resources
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