Gene Therapy Cuts Seizures 85% in Kids with Dravet Syndrome

Twelve-year-old Owen is doing something his mother Austin never thought possible: making friends and playing with other kids his age.

Owen has Dravet syndrome, a severe form of epilepsy that typically begins in infancy and causes frequent seizures along with cognitive delays, communication challenges, and developmental problems. For years, his condition kept him from activities most kids take for granted.

That changed when Owen joined a clinical trial at Ann & Robert H. Lurie Children's Hospital of Chicago testing a new gene therapy called zorevunersen. The treatment works differently from traditional seizure medications by targeting the root cause of Dravet syndrome, a mutation in the SCN1A gene.

The results have stunned researchers. Among 81 children aged 2 to 18 in the Phase 1/2a trial, those who received the higher doses saw their motor seizures drop by 85% at three months and 73% at six months. Kids who continued treatment in follow-up studies maintained seizure reductions between 58% and 90% over 20 months.

But the improvements went beyond seizure control. Children who stayed in the extension studies for more than three years showed significant gains in both understanding and expressing language, skills that often lag severely in Dravet patients.

"He is able to make friends, which is a new and wonderful development," Austin says about Owen. "His quality of life has improved so much that he can now enjoy activities with neurotypical peers."

The treatment enhances the function of the normal copy of the SCN1A gene that Dravet patients still have, compensating for the mutated one. Dr. Linda Laux, who led the trials, says the data supports both the safety of zorevunersen and its ability to improve overall quality of life and adaptive behavior.

The Ripple Effect

For families affected by Dravet syndrome, this breakthrough means more than reduced seizures. It means children who can communicate better, learn more easily, and participate in everyday activities. It means parents who can worry less about their child's next seizure and focus more on helping them thrive. And it represents a shift in how we treat severe epilepsy, moving from managing symptoms to addressing genetic causes.

The findings were published in the New England Journal of Medicine in April 2026, and a Phase 3 trial is already underway to confirm these promising results in a larger group of patients.

For Owen and kids like him, the future just got a whole lot brighter.