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…
News
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…
A Phase 3 clinical trial testing the therapy candidate zorevunersen for Dravet syndrome is now recruiting children who still experience seizures despite being on stable treatment with antiseizure medications. The study, dubbed EMPEROR (NCT06872125), is open to pediatric patients ages 2 to 17. Enrollment is ongoing at…
Daily treatment with Fycompa (perampanel), on top of standard medications, was safe and reduced seizure frequency for children with Dravet syndrome whose seizures had not been previously controlled with other therapies, according to an observational study in China. Fycompa’s benefits were observed in children of all ages, including…
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