Margarida Maia, PhD, science writer —

Encoded Therapeutics has been cleared by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to begin clinical testing of ETX101, an investigational one-time gene therapy for children with Dravet syndrome. A Phase 1/2 clinical study dubbed EXPEDITION will test how safe ETX101 is and how well it…

Health Canada has approved Epidiolex (cannabidiol) as an add-on treatment for seizures in adults and children, ages 2 and older, with Dravet syndrome, expanding the list of countries where the medicine is available for this form of epilepsy. The oral solution developed by GW Pharmaceuticals, now Jazz…

During clinical trials, side effects are more common in people who use cannabidiol (CBD) to treat some forms of epilepsy, including Dravet syndrome, than in people on a placebo, according to a recent study. Those side effects ranged from mild to severe and could be serious enough to make…

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…

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…

Up to 1 in 4 people with hard-to-treat epilepsy, including those with Dravet syndrome, were found to stop taking Epidiolex (cannabidiol) shortly after starting treatment, primarily due to side effects or a lack of efficacy, according to a real-world study. “Epidiolex is generally well-tolerated and the majority continued…

More severe epilepsy and greater behavioral challenges predict worse health-related quality of life (HRQoL) up to 10 years later in people with Dravet syndrome, a study has found. In addition, early use of sodium channel blockers — a type of anti-seizure medication that should be avoided in most…

Biocodex has chosen Pantherx Rare specialty pharmacy to be the sole U.S. distributor of Diacomit (stiripentol), an add-on and oral anti-seizure medicine that is approved for people with Dravet syndrome, ages 2 and older. The pharmacy, licensed in all 50 states, will add Diacomit to its portfolio…

Treatment with soticlestat, an inhibitor of cholesterol turnover in the brain, protected against fever-induced seizures in two mouse models of Dravet syndrome, a study has found. In one of the models, the experimental therapy also prevented sudden unexpected death in epilepsy (SUDEP), a rare complication of seizures —…

Pharmaceutical giant UCB has agreed to buy Zogenix, the developer of Fintepla (fenfluramine), an oral add-on medicine that treats seizures associated with Dravet syndrome, a rare type of epilepsy. The purchase, which could cost UCB up to about $1.9 billion, is a step toward expanding the company’s…