Margarida Maia, PhD, science writer —

In a mouse model study of Dravet syndrome, males were seen to be more vulnerable to abnormal heartbeats and heart deficits in energy-producing mitochondria than female mice. These findings suggest that male Dravet patients may be particularly vulnerable to sudden unexpected death in epilepsy (SUDEP), a fatal seizure…

Stoke Therapeutics’ zorevunersen, a treatment that addresses the genetic cause of Dravet syndrome, continues to reduce the frequency of seizures in children and adolescents who are taking part in Phase 1/2 clinical studies in the U.S. and the U.K., while also leading to improvements in cognition and behavior.

Nine months of treatment with bexicaserin (LP352), an investigational oral small molecule from Longboard Pharmaceuticals, more than halved the frequency of motor seizures in adults and adolescents with Dravet syndrome or other forms of developmental and epileptic encephalopathies (DEE). That’s according to new interim data from the…

Encoded Therapeutics has been cleared by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to begin clinical testing of ETX101, an investigational one-time gene therapy for children with Dravet syndrome. A Phase 1/2 clinical study dubbed EXPEDITION will test how safe ETX101 is and how well it…

Health Canada has approved Epidiolex (cannabidiol) as an add-on treatment for seizures in adults and children, ages 2 and older, with Dravet syndrome, expanding the list of countries where the medicine is available for this form of epilepsy. The oral solution developed by GW Pharmaceuticals, now Jazz…

During clinical trials, side effects are more common in people who use cannabidiol (CBD) to treat some forms of epilepsy, including Dravet syndrome, than in people on a placebo, according to a recent study. Those side effects ranged from mild to severe and could be serious enough to make…

Longboard Pharmaceuticals has finished enrolling patients in PACIFIC, its Phase 1b/2a clinical study of LP352, an investigational oral serotonin receptor agonist for Dravet syndrome and other types of developmental and epileptic encephalopathies (DEEs). With 52 patients enrolled in 30 sites in the U.S. and Australia, the study’s main…

BMB-101, an oral serotonin agonist being developed by Bright Minds Biosciences for the treatment of Dravet syndrome and other forms of epilepsy, was found to be safe and well-tolerated in healthy individuals, even at the highest dose tested. These are data from a now-complete Phase 1 study…

Up to 1 in 4 people with hard-to-treat epilepsy, including those with Dravet syndrome, were found to stop taking Epidiolex (cannabidiol) shortly after starting treatment, primarily due to side effects or a lack of efficacy, according to a real-world study. “Epidiolex is generally well-tolerated and the majority continued…

More severe epilepsy and greater behavioral challenges predict worse health-related quality of life (HRQoL) up to 10 years later in people with Dravet syndrome, a study has found. In addition, early use of sodium channel blockers — a type of anti-seizure medication that should be avoided in most…