A groundbreaking clinical trial at Sheffield Children’s Hospital in the UK has dramatically transformed the lives of children with Dravet syndrome, a rare and severe form of epilepsy. Among the participants are seven-year-olds Albie Kelly and Freddie Truelove, who have experienced remarkable improvements since beginning treatment with the experimental drug zorevunersen, formerly known as STK-001.
Before the trial, both children endured frequent, debilitating seizures that limited their ability to speak, socialize, and live independently. Albie’s mother, Lauren Kelly, described how their lives revolved around the fear of seizures, even avoiding travel to remain close to medical care. Since Albie received his first dose in June 2022, the changes have been extraordinary. Once non-verbal and emotionally withdrawn, Albie now attends school full-time, communicates clearly, plays sports, and shows joy and independence that were previously unimaginable.
Freddie, from near Huddersfield, was the first child under 13 in the UK to receive the drug in September 2021. Before treatment, he suffered more than a dozen seizures each night and had limited speech. Today, his seizures are minimal, occurring just once every few days or not at all for extended periods. He can now walk up stairs, swim, ski, and interact with peers thanks to his improved speech and cognitive abilities. His mother, Lauren Truelove, called the impact “life-changing,” adding that the trial gave them a life they never believed possible.
Led by Stoke Therapeutics, the trial involves an observation phase followed by three doses of the drug over 12 weeks, then a maintenance phase with one dose every 16 weeks. Currently, six children are being treated at Sheffield Children’s, with around 17 participants nationwide.
Consultant Dr. Archana Desurkar praised the children’s progress, noting how Albie now sings and expresses affection, and how Freddie’s skiing trip demonstrated his newfound physical ability and joy. She emphasized that Dravet syndrome is typically associated with treatment-resistant seizures, significant neurological impairment, and even early mortality. This research offers not only hope for the children involved but also future generations.
Although it may take several years for zorevunersen to become widely available due to regulatory processes, its potential to reshape the standard of care for Dravet syndrome is already clear.
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