Steve Bryson, PhD, science writer —

STK-001, an investigational treatment for children and adolescents with Dravet syndrome, markedly reduced seizure frequency and improved cognition and behavior, according to new findings in Phase 1/2a clinical trials. “The substantial and sustained reductions in seizure frequency and improvements in cognition and behavior observed in our STK-001 clinical studies…

Nighttime monitoring devices are used by most Dravet syndrome caregivers — at least 75% — to detect seizures and prevent sudden unexpected death in epilepsy, or SUDEP, according to the results of a questionnaire-based study in Germany. Such monitoring is important because, statistics show, children with Dravet have…

Newer antiseizure medications for children and adolescents with Dravet syndrome outperformed placebos and reduced seizure frequency by at least 50% in clinical trials, according to a pooled analysis of study data. The review also compared the benefits and safety of such therapies, each of which is approved in the…

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…