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Biogen and Stoke report positive results for Dravet syndrome drug

Ella Day | July 17, 2025 | News story | Research and Development Biogen, Dravet Syndrome, Neurology, Stoke Therapeutics, epilepsy, rare diseases 

Biogen and Stoke Therapeutics have shared encouraging new data for their experimental treatment, zorevunersen, which is being developed for Dravet syndrome – a rare, severe form of epilepsy. The results, presented at the European Paediatric Neurology Society Congress in Munich, Germany, showed that patients treated with zorevunersen experienced improvements in cognition and behaviour after a year of treatment.

Dravet syndrome causes seizures along with developmental delays and behavioural challenges. While several anti-seizure medicines are available, most patients still experience seizures, and no approved treatments currently treat the wider developmental problems caused by the condition.

Zorevunersen aims to address underlying causes of the disease. It aims to increase the production of the SCN1A gene, which is faulty in individuals with the disorder and leads to a shortage of a vital brain protein called NaV1.1.

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Previous trials showed that the treatment could significantly reduce seizures. This latest analysis, which helps shape the design of the ongoing phase 3 EMPEROR trial, suggests that it may also improve how young patients learn, communicate and cope with daily activities.

“The data gives us early evidence that this new approach could address the underlying cause of Dravet syndrome, resulting in additional seizure control and offering patients the opportunity to experience improvements in cognition and behaviour,” said Andreas Brunklaus, consultant paediatric neurologist at the Royal Hospital for Children in Glasgow, Scotland.

The phase 3 trial will continue to investigate whether these benefits are confirmed in a larger group of patients.

Ella Day

17/7/25

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