Andrea Lobo, PhD, science writer —

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…