Unmet Needs and the Impact of Disease Modifying Therapies in Dravet Syndrome

In “Unmet Needs and the Impact of Disease Modifying Therapies in Dravet Syndrome,” the panelists explore the limitations of current treatment approaches and discusses how emerging disease-modifying therapies may transform the future management of Dravet syndrome. Expert faculty review the significant advances achieved with currently available antiseizure medications while acknowledging that substantial unmet needs remain for many patients and families.

The discussion highlights that existing therapies are primarily designed to reduce seizures and seizure-related complications, including status epilepticus and sudden unexpected death in epilepsy (SUDEP). Although these treatments have improved outcomes and quality of life for many patients, more than half of individuals with Dravet syndrome continue to experience significant seizure burden despite current standards of care. In addition, panelists emphasize that existing therapies do not directly address many of the cognitive, developmental, behavioral, motor, and sleep-related challenges that contribute to long-term disability and caregiver burden.

Expert faculty then explore how disease-modifying therapies may address these gaps by targeting the underlying genetic cause of disease rather than treating seizures alone. The panel reviews evidence from preclinical models demonstrating the potential benefits of restoring SCN1A function and discusses lessons learned from other genetic neurologic disorders, including spinal muscular atrophy, where earlier intervention has been associated with improved long-term outcomes. Throughout the conversation, panelists emphasize the growing importance of early diagnosis and genetic testing as the field moves toward precision medicine. Faculty discuss how disease-modifying approaches may ultimately provide opportunities to influence both seizure outcomes and the broader neurodevelopmental manifestations of Dravet syndrome, potentially altering the course of disease rather than simply managing its symptoms.

The next episode in this series, “Emerging Therapies in Dravet Syndrome,” features the panelists discussing the clinical relevance of early efficacy and safety findings from investigational therapies, including zorevunersen and ETX101. The expert faculty examine how these approaches may influence both seizure outcomes and broader developmental trajectories in patients with Dravet syndrome.