A new drug, zorevunersen, is showing promising results in treating children with Dravet syndrome, a severe form of epilepsy, according to doctors and families.
Dravet syndrome affects about one in every 15,000 babies born and can cause dozens of dangerous seizures a day. The new drug, administered into the spine via an infusion, works by managing the underlying cause in most cases - a faulty gene affecting the brain.
Freddie Truelove, from Huddersfield, is one of the first children in the UK to receive the new treatment and has gone from having hundreds of seizures a day to a couple a week, as reported by BBC News.
Treatment Results
The early trial results, published in the New England Journal of Medicine, show the experimental treatment can be given safely to adolescents and young children, from the age of two onwards. Young patients in trials in the US and the UK had up to 90% fewer seizures while on repeat doses of the new medication.
Prof Helen Cross, one of the lead researchers from University College London's Institute of Child Health and Great Ormond Street Hospital, says the results have been truly promising. "It is exciting. It's amazing. With improvements, that gives them real hope that they are able to carry out more normal lives, particularly with their families."
Expert Reaction
Dravet Syndrome UK chair of trustees Galia Wilson said: "We regularly see the devastating impact that this condition has on the lives of families. That's why we're so thrilled about these latest results from the initial zorevunersen clinical trials."
More results over the coming years are needed to check how effective the treatment is before it can become widely recommended. However, experts say it offers real hope for families living with Dravet syndrome.
