Scientists laud life-changing drug for children with resistant form of epilepsy
#Zorevunersen #Dravet syndrome #epilepsy treatment #genetic disorders #clinical trials #UCL #Great Ormond Street Hospital #seizure reduction
📌 Key Takeaways
- Zorevunersen reduces seizures by up to 80% in children with Dravet syndrome
- The drug improves quality of life, including motor skills and communication abilities
- Phase 3 trials planned to study long-term effects and identify which patients benefit most
- Treatment targets underlying genetic cause rather than just managing symptoms
- Could potentially be adapted for other forms of genetic epilepsy
📖 Full Retelling
Scientists at University College London and Great Ormond Street Hospital have announced promising preliminary trial results for Zorevunersen, a potentially life-changing drug for children with Dravet syndrome, a rare and treatment-resistant form of epilepsy affecting approximately 3,000 people in the UK. The preliminary trials involved 81 children aged between two and 18 who had Dravet syndrome, a genetic disorder characterized by treatment-resistant epilepsy and often accompanied by speech and developmental delays. Before the study, participants experienced an average of 17 seizures per month, but after taking a 70mg dose of Zorevunersen, they had on average 50% fewer seizures, with about 80% fewer seizures after three doses. The study, published in The New England Journal of Medicine, also demonstrated improved quality of life, including enhanced motor skills, communication abilities, and coping mechanisms. Lead author Helen Cross emphasized the significance of these findings for patients with hard-to-treat genetic epilepsies who often require around-the-clock care and face high risks of sudden unexpected death in epilepsy, with a phase 3 clinical trial now planned to study long-term effects and identify which patients benefit most.
🏷️ Themes
Medical Breakthrough, Genetic Disorders, Pediatric Treatment, Clinical Research
📚 Related People & Topics
Dravet syndrome
Genetic form of epilepsy
Dravet syndrome (DS), previously known as severe myoclonic epilepsy of infancy (SMEI), is an autosomal dominant genetic disorder which causes a catastrophic form of epilepsy, with prolonged seizures that are often triggered by hot temperatures or fever. It is very difficult to treat with anticonvuls...
Great Ormond Street Hospital
Children's hospital in London, England
Great Ormond Street Hospital (informally GOSH, formerly the Hospital for Sick Children) is a children's hospital in the London Borough of Camden, and a part of Great Ormond Street Hospital for Children NHS Foundation Trust. The hospital is situated between Great Ormond Street and Guilford Street, ju...
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Original Source
Scientists laud life-changing drug for children with resistant form of epilepsy Preliminary trials into Zorevunersen find drug to be safe and well tolerated by those with Dravet syndrome Scientists have hailed a potentially life-changing drug for children with a hard to treat form of epilepsy, after promising early clinical trial results. Dravet syndrome is a genetic disorder which causes treatment resistant epilepsy and is often accompanied by speech and developmental delays. About 3,000 people are thought to have the condition in the UK. Current treatments aim to control the number and severity of seizures, but often do not work. These preliminary trials, led by UCL and Great Ormond Street hospital , found that the drug appeared to be safe and well tolerated by the 81 children taking part. Before the study, the participants – aged between two and 18 – experienced an average of 17 seizures a month. But after taking a 70mg does of Zorevunersen, they had on average 50% fewer seizures, and about 80% fewer seizures after three doses. The study, published in The New England Journal of Medicine , also showed improved quality of life, including motor skills, communication and ability to cope. A phase 3 clinical trial will be conducted to study Zorevunersen over time, to identify possible long-term risks and any rare but potentially serious side effects, and to determine which patients are most likely to benefit. The lead author Helen Cross, director and professor of childhood epilepsy at the UCL Institute of Child Health and honorary consultant in paediatric neurology at GOSH, said: “I regularly see patients with hard-to-treat genetic epilepsies, who can have multiple seizures a week. Many are unable to do anything independently for themselves; they require around the clock care and are at high risk of sudden expected death in epilepsy.” If the phase 3 trials are successful, she added, this new treatment “could help children with Dravet syndrome lead much healthier and ha...
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