A new drug called zorevunersen, developed by Biogen and Stoke Therapeutics, has shown potential to be the first treatment that can change the course of a severe form of epilepsy called Dravet syndrome. This news comes from recent clinical trials. Dravet syndrome is a rare and hard-to-treat form of epilepsy that usually starts in childhood. Current treatments often don’t work well for most patients. Zorevunersen is an antisense oligonucleotide treatment, which is a type of medicine that can help correct problems at the genetic level. In the initial trials, 81 children were given the drug. Some received a single dose, while others received multiple doses over six months. The results were promising: the number of seizures decreased by 59% to 91% over the first 20 months, and this improvement lasted for up to three years. This is a significant achievement, as current treatments often fail to control seizures in Dravet syndrome patients. Zorevunersen works by helping to restore normal function to nerve cells, addressing the root cause of the disease. Dr. Barry Ticho, Chief Medical Officer of Stoke Therapeutics, said that this drug could be the first to modify the course of Dravet syndrome. Professor Helen Cross, who led the research, added that these results could mark a turning point in treating Dravet syndrome. The improvements in seizures, as well as in cognition, behavior, and quality of life, suggest that the drug might be changing the course of the disease. Zorevunersen is currently being tested in a larger phase III study, with results expected in mid-2027. The recent findings were published in the New England Journal of Medicine.