– ISTOCK/ THITAREESARMKASAT – Archivo
Madrid, March 6, 2026 –
A new experimental treatment is showing significant promise for children with a severe form of epilepsy, offering a potential path toward drastically reduced seizures and improved quality of life. The findings come from an international clinical trial led by University College London (UCL) and Great Ormond Street Hospital in the United Kingdom.
Published in the New England Journal of Medicine, the research revealed that children with Dravet syndrome experienced up to a 91 percent reduction in seizures when regularly administered a novel medication called zorevunersen. This is particularly significant as effective treatments for Dravet syndrome have been historically limited.
Importantly, the study also indicated a potential for the treatment to lessen the disease’s impact on cognitive and behavioral processes – an area often overlooked in epilepsy care. Over a three-year period, researchers observed improvements in the children’s overall quality of life, and most side effects associated with the treatment were mild.
Dravet syndrome is a devastating genetic condition characterized by frequent, difficult-to-control seizures, and long-term neurological developmental delays. Children with the syndrome often experience feeding difficulties, movement problems, and face an elevated risk of premature death. Current treatments often fail to adequately control seizures, and no approved medications specifically address the cognitive and behavioral challenges associated with the condition.
Zorevunersen, developed by Stoke Therapeutics in collaboration with Biogen, targets the underlying genetic cause of the disease. Most individuals with Dravet syndrome have a defective copy of the SCN1A gene, which is crucial for proper nerve cell function. The medication works by increasing levels of the protein produced by the healthy SCN1A gene, aiming to restore normal nerve cell activity.
Researchers have now released the latest results from their initial trial and ongoing extension studies, which included 81 children with Dravet syndrome from the United Kingdom and the United States.
The published data comes from initial studies designed primarily to assess the safety and tolerability of zorevunersen. Researchers also evaluated the drug’s effects on seizure frequency, cognitive function, behavior, and quality of life. A phase three trial is currently underway to further evaluate the treatment.
“I regularly see patients with difficult-to-treat genetic epilepsies, with consequences that extend far beyond seizures, and it’s heartbreaking to see limited treatment options,” said Professor Helen Cross, lead author and Director and Professor of Childhood Epilepsy at UCL’s Institute of Health and honorary consultant in Paediatric Neurology at Great Ormond Street Hospital (GOSH).
“This new treatment has the potential to help children with Dravet syndrome live much healthier and happier lives. Our findings demonstrated that zorevunersen is safe and well-tolerated by the majority of patients, justifying further evaluation in the ongoing phase three trial.”
A total of 81 children, aged between two and eighteen years, participated in the initial trial. Before the start of the trials, these patients experienced an average of 17 seizures per month. The children received up to 70 mg of zorevunersen via lumbar puncture, either as a single dose or with additional doses two or three months later over a six-month period. Of these patients, 75 continued to receive the drug every four months in extension studies.
Patients who received a 70 mg dose in the initial phase of the trial saw their seizures reduced by 59 to 91 percent during the first 20 months of the extension studies, compared to their seizure frequency before the trial began.
Nineteen of the trial participants were patients at hospitals across the United Kingdom, including GOSH, Sheffield Children’s Hospital, Evelina London Children’s Hospital, and Royal Hospital for Children, Glasgow.
At GOSH, the trial was conducted at the Clinical Research Facility of the National Institute for Health and Care Research, a state-of-the-art facility dedicated to children participating in experimental trials.
Galia Wilson, Chair of the Advisory Board for Dravet Syndrome UK, stated: “We frequently witness the devastating impact this condition has on families’ lives. That’s why we are so encouraged by these latest results from the early clinical trials of zorevunersen. We now look forward to the phase three clinical trials to see if the initial promise we see here translates into real hope for all families currently affected by Dravet syndrome.”
