Experimental drug zorevunersen reduces seizures by ninety one percent in children with rare dravet syndrome

UCL researchers find zorevunersen significantly reduces seizures in children with Dravet syndrome by boosting protein from the SCN1A gene.

By: AXL Media

Published: Mar 5, 2026, 3:22 AM EST

Source: The information in this article was sourced from University College London

Breakthrough in Genetic Epilepsy Treatment

An international clinical trial has revealed that an experimental therapy called zorevunersen is both safe and highly effective for children suffering from Dravet syndrome. This rare and severe form of genetic epilepsy is characterized by frequent, difficult to control seizures and long term neurodevelopmental challenges. Led by researchers from University College London and Great Ormond Street Hospital, the study published in The New England Journal of Medicine found that some participants experienced a reduction in seizures of up to 91 percent. These findings represent a significant shift in the treatment landscape for a condition that has historically lacked targeted therapeutic options.

Mechanism of Action for Zorevunersen

Zorevunersen, developed by Stoke Therapeutics in collaboration with Biogen, is designed to address the root cause of the disorder rather than merely masking symptoms. Most individuals possess two copies of the SCN1A gene, which is responsible for producing a protein essential for healthy nerve cell signaling. In those with Dravet syndrome, one of these copies is faulty and fails to produce sufficient protein levels. The new drug works by stimulating the remaining healthy copy of the SCN1A gene to increase its protein output. By restoring these protein levels toward a normal range, the therapy aims to stabilize nerve cell function and prevent the electrical surges that lead to seizures.

Clinical Trial Data and Seizure Reduction

The trial involved 81 children between the ages of two and 18 who initially experienced an average of 17 seizures per month. Participants received doses of up to 70mg of the drug via lumbar puncture. During the initial six month period and subsequent extension studies lasting up to 20 months, researchers observed dramatic improvements. Among those receiving the highest dose, seizure frequency dropped between 59 percent and 91 percent compared to their baseline levels before the trial. Furthermore, the majority of reported side effects were mild, suggesting that the treatment is well tolerated over long periods.