Marisa Wexler, MS,  senior science writer—

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…

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…

June is Dravet Syndrome Awareness Month in the U.S., and the Dravet Syndrome Foundation is spearheading efforts to raise funds and spread the word about this rare type of epilepsy. “By raising awareness of Dravet syndrome, our community can increase knowledge in the general public, improve early diagnosis,…

Praxis Precision Medicines is gearing up to launch a clinical trial of its experimental anti-seizure therapy relutrigine in people with Dravet syndrome and other disorders that cause seizures in childhood, collectively known as developmental and epileptic encephalopathies (DEEs). According to a corporate presentation, the study, dubbed EMERALD,…

Stoke Therapeutics and Biogen are teaming up for the further development and potential commercialization of zorevunersen, an experimental treatment for Dravet syndrome that’s moving into late-stage clinical testing. “With Biogen’s deep experience in neurology and track record of success in commercializing high-value disease-modifying medicines for…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to relutrigine, a treatment candidate for Dravet syndrome that’s being developed by Praxis Precision Medicines. The therapy aims to reduce seizures in children with epileptic disorders such as Dravet. “We are thrilled to have been…

Targeting a heart protein called NaV1.6 may help prevent sudden death in adults with Dravet syndrome, a study in mice suggests. The study, “Cardiac-Specific Deletion of Scn8a Mitigates Dravet Syndrome-Associated Sudden Death in Adults,” was published in JACC: Clinical Electrophysiology. Dravet syndrome is caused by…

The U.S. Food and Drug Administration (FDA) has lifted its partial clinical hold on higher doses of zorevunersen, an experimental treatment for Dravet syndrome currently being tested in Phase 1/2 trials. The FDA placed the hold based on preclinical data in 2020, limiting dosing of zorevunersen in studies taking…

Parents of children with Dravet syndrome in Spain say they don’t get enough support to help meet the emotional and economic challenges of the disease, a study found. “The findings of this study underscore the need for policy reforms that specifically address the challenges faced not only by individuals…

Although high-quality data are lacking, available evidence suggests that a procedure called vagus nerve stimulation (VNS) — which targets a large nerve connecting the brain and the gut — can help to reduce seizure frequency in some people with Dravet syndrome. That’s according to a new study titled…