News

Treatment with the approved Dravet syndrome drug Fintepla (fenfluramine) was more effective than a placebo at reducing seizure frequency and extending periods without seizures among a diverse group of children and adolescents with the rare disease, data from three late-stage clinical trials show. The oral therapy, widely cleared…

Encoded Therapeutics has raised $275 million in Series F financing to support the clinical development of its experimental gene therapy ETX101 for infants and young children with Dravet syndrome. The funds will also support an expansion study in children and adolescents up to age 18, and the commercial…

Treatment with the experimental therapy zorevunersen may alter the progression of Dravet syndrome, according to new long-term data from early clinical trials. The data indicate that zorevunersen led to long-term reductions in seizure rates, improved communication abilities, and overall quality of life, and that the experimental treatment was generally…

Children with Dravet syndrome may experience multiple seizures before receiving a diagnosis and starting disease-specific treatment, with substantial delays reported between seizure onset, genetic testing, diagnosis, and treatment, a European survey study shows. Among 45 patients whose diagnostic journeys were reviewed, seizures began at a median age of 4…

Newer add-on anti-seizure medications may help reduce the burden of life-threatening status epilepticus in people with Dravet syndrome, a real-world study suggests. Fintepla (fenfluramine) was associated with a lower rate of status epilepticus, while Diacomit (stiripentol) was associated with shorter episodes. Each additional newer anti-seizure medication…

A one-time treatment designed to boost the activity of a gene deficient in most people with Dravet syndrome significantly reduced seizures — both fever-triggered and spontaneous — in a mouse model of the rare condition, a severe form of epilepsy that typically begins in infancy. Specifically, treated mice saw…

Inhibiting a specific immune signaling pathway in the brain may reduce inflammation and seizure susceptibility, making it a potential new treatment target for Dravet syndrome, according to a study in mice. The study led by Li Gan, PhD, a professor at Weill Cornell Medical College in New York, found…

Patients will be randomly assigned to groups in a Phase 3 clinical trial testing bexicaserin for seizures associated with Dravet syndrome, according to the treatment’s developer. Lundbeck said it expects to complete patient randomization — assigning participants to groups receiving the treatment or a placebo — within…

Across a group of children with severe childhood epilepsies, including Dravet syndrome, genetic test results did not independently predict long-term seizure or developmental outcomes, according to a new study. “Although … genetic testing has diagnostic and precision-medicine selection utility in childhood-onset [developmental and epileptic encephalopathies], long-term seizure and developmental…

The first child has received ION337 in an open-label Phase 1/2 study, marking the start of clinical testing of this experimental therapy for Dravet syndrome. The study, called ASCEND (NCT07531745), is recruiting up to 32 children, ages 2 to 12, who have been diagnosed with Dravet syndrome and…