News

Fintepla (fenfluramine) significantly reduces seizure frequency in children and adolescents with Dravet syndrome who experience hard-to-treat seizures, according to new Phase 3 trial data. These findings add to positive results from other Fintepla Phase 3 trials, further supporting the benefits of the approved add-on therapy for this…

Longboard Pharmaceuticals has finished enrolling patients in PACIFIC, its Phase 1b/2a clinical study of LP352, an investigational oral serotonin receptor agonist for Dravet syndrome and other types of developmental and epileptic encephalopathies (DEEs). With 52 patients enrolled in 30 sites in the U.S. and Australia, the study’s main…

Epidiolex (cannabidiol) reduces the frequency of seizures for most people with Dravet syndrome and also leads to gains in cognitive function, communication, and behavior for many patients, according to a study based on interviews with caregivers. “Caregivers’ Perspectives on the Impact of Cannabidiol (CBD) Treatment for Dravet…

BL-001, an investigational oral therapy for Dravet syndrome, was well-tolerated across multiple-ascending doses in healthy volunteers, top-line data from a Phase 1 trial show. These results indicate that BL-001 can be dosed at concentrations up to 10-fold higher than the most effective dose in animal models, according to…

BMB-101, an oral serotonin agonist being developed by Bright Minds Biosciences for the treatment of Dravet syndrome and other forms of epilepsy, was found to be safe and well-tolerated in healthy individuals, even at the highest dose tested. These are data from a now-complete Phase 1 study…

Treatment with STK-001 led to a marked reduction in seizure frequency, and aided cognition and behavior, among children and adolescents with Dravet syndrome in early clinical trials, according to new findings announced by the therapy’s developer Stoke Therapeutics. “Together these data support the potential for STK-001 to…

Baylor Genetics is offering a gene analysis service on epileptic disorders, including conditions like Dravet syndrome. The specialized panel is designed for outpatient use and is intended to assist clinicians in assessing the genetic factors underlying a patient’s epilepsy. “When a genetic cause of epilepsy is suspected, a…

A novel gene therapy developed by a team of scientists was able to reduce seizures, improve cognitive scores, and extend survival in a mouse model of Dravet syndrome, a new study reports. The therapy showed beneficial effects even when administered after the animals had already begun experiencing seizures. “The…

Researchers have created a new rat model of Dravet syndrome that captures early features of the disease that may make it more suitable for long-term studies than other models. The study, “A novel rat model of Dravet syndrome recapitulates clinical hallmarks,” was published in Neurobiology of…

Epidyolex may be safe and effective for people with Dravet syndrome and Lennox-Gastaut syndrome (LGS) without accompanying use of clobazam (brand names Onfi and Sympazan), according to a recent European analysis. While the oral cannabidiol solution is cleared for use without clobazam in the U.S. — where it…