Andrea Lobo, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to Longboard Pharmaceuticals‘ bexicaserin to treat seizures associated with developmental and epileptic encephalopathies (DEEs), including Dravet syndrome, in patients 2 and older. The designation is meant to accelerate the development and regulatory review of medications intended to…

Epidiolex (cannabidiol) can help to reduce seizure frequency in some adults with generalized or localized forms of epilepsy — including Dravet syndrome — that are resistant to other anti-seizures medications, according to a retrospective study in France. Among the 27% of patients who responded to this treatment, most…

Epygenix Therapeutics, the company developing EPX-100 (clemizole) as a potential add-on treatment for people with Dravet syndrome, has been acquired by Harmony Biosciences. “This acquisition builds on our leadership position in sleep/wake and our franchise in neurobehavioral disorders, allowing us to leverage our expertise in CNS…

Encoded Therapeutics has received clearance from U.S. and Australian regulatory agencies to launch Phase 1/2 clinical trials testing ETX101, its Dravet syndrome gene therapy candidate, in infants and young children with the seizure disorder. In the U.S., the company plans to begin ENDEAVOR (NCT05419492), which will be…

Adding Fintepla (fenfluramine) to anti-seizure treatment is associated with a more than five times higher chance of having a 50% decline in the frequency of Dravet syndrome-related seizures, a review of clinical trial data shows, Decreased appetite, diarrhea, fatigue, and weight loss were among the noncardiovascular side effects…

Adopting a ketogenic diet — one low in carbohydrates and high in fat — safely and effectively reduces the frequency of seizures and improves cognitive function and behavior in children and adolescents with Dravet syndrome, according to a multicenter study in China. Nearly a third of patients were…

Brain-like organoids, or “mini-brains,” that are specific to affected brain regions and derived from patients could act as models for studying the underlying molecular mechanisms of Dravet syndrome, a study shows. Researchers created patient-derived organoids of the forebrain, a region enriched with types of nerve cells called GABAergic neurons…

Fintepla (fenfluramine) is associated with dose-dependent, clinically meaningful improvements in everyday behavioral and emotional control, as well as the ability to adapt to new situations, in preschool-aged children with Dravet syndrome. These benefits weren’t always tied to a clinically meaningful reduction in the frequency of seizures, suggesting the…