Gene Regulation Therapy Shows Promise for Dravet Syndrome

Early-phase clinical trials of a new gene-regulation drug demonstrate significant improvements in seizure control and other symptoms for patients with the rare epilepsy disorder.

Mar. 11, 2026 at 8:02am

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A first-ever gene-regulation therapy tested in early-phase clinical trials co-led by Northwestern University Feinberg School of Medicine and Ann & Robert H. Lurie Children's Hospital of Chicago has proven safe and effective for patients with Dravet syndrome, a rare and severe form of epilepsy. The drug, called zorevunersen, delivered striking improvements in seizure frequency, language, motor skills and behavior, marking a potential turning point for patients whose conditions have long resisted standard treatment.

Why it matters

Dravet syndrome is a devastating condition that emerges in infancy, causing seizures, cognitive deficits, communication and behavioral impairments, motor dysfunction, growth delays and autistic traits. Current treatment options are limited, so these promising results from the gene-regulation therapy trials offer new hope for families struggling with this rare disorder.

The details

The two Phase 1/2a, open-label, multicenter studies enrolled 81 patients with Dravet syndrome aged 2-18 years. Patients who received two to three doses of 70 mg zorevunersen had a reduction of motor seizures of nearly 85% at three months and 73% at six months. Eligible patients then transitioned to open-label extension studies, where they continued to see significant seizure reduction ranging from 58% to 90% over the first 20 months. Patients in the extension studies for more than 36 months also showed significantly improved expressive and receptive communication.

  • The results were published last week in the New England Journal of Medicine.
  • A Phase 3, double-blind, placebo-controlled trial of zorevunersen for Dravet syndrome is currently underway.

The players

Linda Laux

Head of the Epilepsy Center, an associate professor of pediatrics at Feinberg and the associate division head of neurology at Lurie Children's, who co-led the clinical trials.

Owen

A 12-year-old Lurie Children's patient with Dravet syndrome who participated in the clinical trial and continues in the open-label extension study. With zorevunersen, Owen's seizures are significantly reduced, and he has had marked improvement in language and behavior.

Austin

Owen's mother, who said his quality of life has increased substantially and he is now able to enjoy more activities with neurotypical peers.

Stoke Therapeutics

The company that provided funding for the clinical trials.

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What they’re saying

“Our results are highly promising, especially since currently there are no approved treatments that address the underlying cause of Dravet syndrome. Since this gene regulation product targets the actual root cause of Dravet syndrome, we observed improvements in other developmental and cognitive symptoms, in addition to seizure control. This is unprecedented.”

— Linda Laux, Head of the Epilepsy Center, associate professor of pediatrics at Feinberg, and associate division head of neurology at Lurie Children's (New England Journal of Medicine)

“He is able to make friends, which is kind of a new development. His quality of life has increased substantially so that he's able to enjoy more activities with neurotypical peers.”

— Austin, Owen's mother (Instagram)

What’s next

A Phase 3, double-blind, placebo-controlled trial of zorevunersen for Dravet syndrome is currently underway.

The takeaway

This gene-regulation therapy represents a potential breakthrough for families dealing with the devastating effects of Dravet syndrome, offering new hope for improved seizure control and quality of life for patients who have long struggled with this rare and severe form of epilepsy.