Abstract
Dravet's syndrome (DS) or severe myoclonic epilepsy of infancy is a rare, genetic,
and infantile-onset epileptic encephalopathy. DS presents with recurrent febrile seizures
and/or febrile status epilepticus in developmentally normal infants, and subsequently
evolves into a drug-resistant mixed-seizure disorder with developmental arrest or
regression. As many defining clinical features of DS do not become evident until 3
to 4 years of age, diagnosis is often delayed. Early seizure control, particularly
the prevention of status epilepticus in infancy, has been shown to correlate with
better long-term outcomes. Thus, early diagnosis and seizure control is crucial. Several
treatment algorithms have been published in recent years to guide antiepileptic drug
selection and escalation. Last year, two agents, stiripentol and cannabidiol, were
approved by the U.S. Food and Drug Administration specifically for use in DS, and
a third has been submitted (fenfluramine). Additional therapies, including serotonin
modulators lorcaserin and trazodone, verapamil, and several first-in-class medications,
are currently in various phases of investigation.
Keywords
Dravet syndrome - cannabidiol - fenfluramine
