Overview of Dravet syndrome and an update in therapeutics

2024-03-10T14:57:19+07:00

Dravet syndrome is a developmental epileptic encephalopathy characterized by early onset drug-resistant seizures and other clinical features, including intellectual disability and impairments in behavior, motor, and sleep which cause poor quality of life and impact the long-term course. The pathogenesis of Dravet syndrome is strongly connected to the dysfunction of the voltage-gated sodium channel, more specifically, to mutations in SCN1A gene. Epilepsy in Dravet syndrome is very refractory and seizures remain poorly controlled even with multiple conventional anti-epileptic drugs. Therapeutic approaches in Dravet syndrome have undergone tremendous changes in the lastest years and the current consensus of seizure management strategy in Dravet syndrome consists of a combination of conventional drugs and three recently approved drugs including stiripentol, cannabidiol, and fenfluramine. This review article aims to update recent changes to the therapeutic landscape for Dravet syndrome by summarizing the most key data from the clinical development of stiripentol, cannabidiol, and fenfluramine. This article also reviews and discusses the latest findings of other pharmacotherapies in development, including serotonergic agents, soticlestat, verapamil as well well strategies to treat the underlying cause of Dravet syndrome, including gene therapy and antisense oligonucleotides.

Science and Technology Development Journal: Health Sciences

Overview of Dravet syndrome and an update in therapeutics