Steve Bryson, PhD, science writer —

Note: This article was updated April 21, 2023, to reflect that Fintepla was originally classified a Schedule IV controlled substance due to its low risk of abuse or misuse. A risk evaluation and mitigation strategy established by Zogenix that limits Fintepla’s access due to links with cardiovascular-related side effects remains…

A higher single dose of STK-001 will be administered in the U.S.-based Phase 1/2a MONARCH trial that is testing Stoke Therapeutics’ experimental therapy in children and adolescents with Dravet syndrome. The U.S. Food and Drug Administration (FDA) allowed Stoke to test a 70 mg dose in the MONARCH…

New safety and efficacy data from two Phase 1/2a clinical trials testing STK-001, Stoke Therapeutics’ investigational treatment for children and adolescents with Dravet syndrome, are expected as early as mid-year. Interim results from the ongoing Phase 1/2a MONARCH (NCT04442295) and ADMIRAL (ISRCTN99651026) trials, reported late last…

Restoring the activity of Scn1a — the mouse variant of the gene that often is defective in humans with Dravet syndrome — suppressed seizures and normalized behavior in mice, even after symptom onset, a study showed. Notably, the improvements occurred in juvenile mice as well as in adult mice…

More than 70% of Dravet syndrome (DS) patients demonstrated a reduction in seizure frequency of at least 50% while treated with Fintepla (fenfluramine) as an add-on therapy for up to one year, according to an analysis of real-world data. The antiseizure response to this medication was similar in…

The prevalence of Dravet syndrome in the U.K. is low, with most patients diagnosed before age 18, a large-scale healthcare database analysis showed. The findings also showed that the use of anti-seizure medications and healthcare resources was high among those with Dravet. This study provided valuable information on the…

An international panel of physicians and caregivers with expertise in Dravet syndrome is recommending genetic testing in children ages 2 to 15 months who develop normally but have a prolonged seizure after fever or vaccination. Among several consensus statements, the panel also recommended a number of currently available therapies…

Treatment for one year or longer with the antiseizure therapy Fintepla (fenfluramine) was shown to have a minimal impact on height and weight in children with Dravet syndrome, according to data from an open-label extension study. Despite Fintepla’s active ingredient being fenfluramine — a medicine initially…

Virpax Pharmaceuticals has acquired exclusive worldwide rights from Nanomerics to develop a nasal spray of cannabidiol to treat epilepsy in adults and children, including those with Dravet syndrome. Under the agreement, Virpax can develop, manufacture, and sell VRP324, an investigational formulation of cannabidiol (CBD) administered using a nasal…

Researchers have developed a mathematical model to identify electrical signal patterns in the brain that predict epileptic seizures in advance, of benefit to people with seizure-related disorders such as Dravet syndrome, a study reports.  The study, “…