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Behavioral and social difficulties are common in young children with Dravet syndrome, often emerging in toddlerhood and worsening through early childhood, according to a natural history study. The ENVISION (NCT04537832) study followed 58 children with Dravet syndrome, aged 6 months to 5 years, for up to two years.

An online occupational therapy program that coaches parents on how to help their children problem-solve may help young people with Dravet syndrome improve at daily tasks, a small study suggested. The program is based on the Cognitive Orientation to daily Occupational Performance (CO-OP) Approach, a therapy usually delivered directly…

Lab-grown nerve cells from people with Dravet syndrome have abnormal levels of chemical elements that regulate nerve cell function, including potassium, copper, and zinc, a study found. Researchers said these imbalances may contribute to abnormal brain electrical activity, which makes people with Dravet prone…

Epidiolex (cannabidiol) reduced seizure frequency in five children with Dravet syndrome caused by 2q24.3 microdeletions, according to a case series. Dravet syndrome is a severe form of epilepsy that begins in early infancy. A 2q24.3 microdeletion involves the loss of a segment of chromosome 2 that includes the…

Enrollment in a large clinical trial evaluating zorevunersen for Dravet syndrome is now expected to be completed in the second quarter of this year, earlier than previously anticipated, positioning the therapy candidate for a potential new drug application (NDA) submission in 2027. That’s according to Stoke Therapeutics, which is…

ETX101, an experimental one-time gene therapy for Dravet syndrome, has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA). The FDA grants this designation to investigational treatments for serious conditions that, based on early evidence, may offer a meaningful improvement over existing options. The designation…

ETX101, an investigational one-time gene therapy being developed by Encoded Therapeutics for children with Dravet syndrome, has shown a favorable safety profile and early signs of benefit in an ongoing clinical program. Called the POLARIS program, the clinical effort includes three Phase 1/2 trials of ETX101: ENDEAVOR (NCT05419492)…

Stoke Therapeutics plans to meet with the U.S. Food and Drug Administration (FDA) before the end of this year to review four years of safety and efficacy data on its experimental therapy zorevunersen for Dravet syndrome, and to discuss faster ways to make the treatment available to patients…

The U.S. Food and Drug Administration (FDA) has granted ETX101, an experimental gene therapy for Dravet syndrome, its regenerative medicine advanced therapy (RMAT) designation. According to developer Encoded Therapeutics, the RMAT status was awarded following positive clinical trial data. The regulatory designation is given to certain types of…

Zorevunersen continues to show durable reductions in seizure frequency — alongside gains in cognition, behavior, and day-to-day functioning — in children and adolescents followed for up to three years. The investigational therapy targets the genetic cause of Dravet syndrome. The findings come from two long-term extension studies that followed…