المتخصصون في المجالات الطبية

March 24, 2026

Multicenter research in which Mayo Clinic participated found that a gene therapy has potential to significantly reduce seizure burden in children with Dravet syndrome. As described in the New England Journal of Medicine, the research results suggest that the therapy also can improve overall clinical status, quality of life and adaptive behavior.

Dravet syndrome is a severe epilepsy caused primarily by genetic variants that result in reduced expression of the voltage-gated ion channel known as NaV1.1. The risk of sudden unexpected death in epilepsy is higher among patients with Dravet syndrome than in the general population of patients with epilepsy. The current standard of care — antiseizure medications, dietary therapy and neuromodulation — doesn't lead to seizure control in most patients. The effects on nonseizure symptoms, such as cognitive and communication deficits, are minimal. Disease-modifying therapies that directly target the underlying channelopathy are needed.

Zorevunersen is an antisense oligonucleotide developed to target that channelopathy. In open-label phase 1-2a clinical trials, patients ages 2 to 18 with Dravet syndrome were given the therapy in either a single-ascending dose or a multiple-ascending dose over three months. Eligible patients for rollover to two open-label extension studies continued to receive zorevunersen every four months.

Key findings:

• Patients who received 70 mg of zorevunersen in the phase 1-2a studies, followed by up to 45 mg in the extension studies, had a median decline from baseline in seizure frequency of 58.8% to 90.2% during the first 20 months of the extension studies.
• Most treatment-related adverse events were mild or moderate, the most common being elevated protein levels in cerebrospinal fluid.
• Treatment appeared to be associated with improvement in nonseizure symptoms, including expressive and receptive communication.

"Caregivers have indicated that alleviation of nonseizure symptoms is important for new treatments of Dravet syndrome," says Elaine C. Wirrell, M.D., a pediatric neurologist and director of the Pediatric Epilepsy Program at Mayo Clinic in Rochester, Minnesota, and a co-author of the zorevunersen paper. "Overall, the data from our studies support the potential for disease modification with this gene therapy."

A phase 3, randomized, controlled trial of zorevunersen is underway.

For more information

Laux L, et al. Zorevunersen in children and adolescents with the Dravet syndrome. New England Journal of Medicine. 2026;394:969.

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