Treatment with the experimental therapy zorevunersen may alter the progression of Dravet syndrome, according to new long-term data from early clinical trials. The data indicate that zorevunersen led to long-term reductions in seizure rates, improved communication abilities, and overall quality of life, and that the experimental treatment was generally…
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Children with Dravet syndrome may experience multiple seizures before receiving a diagnosis and starting disease-specific treatment, with substantial delays reported between seizure onset, genetic testing, diagnosis, and treatment, a European survey study shows. Among 45 patients whose diagnostic journeys were reviewed, seizures began at a median age of 4…
Newer add-on anti-seizure medications may help reduce the burden of life-threatening status epilepticus in people with Dravet syndrome, a real-world study suggests. Fintepla (fenfluramine) was associated with a lower rate of status epilepticus, while Diacomit (stiripentol) was associated with shorter episodes. Each additional newer anti-seizure medication…
A one-time treatment designed to boost the activity of a gene deficient in most people with Dravet syndrome significantly reduced seizures — both fever-triggered and spontaneous — in a mouse model of the rare condition, a severe form of epilepsy that typically begins in infancy. Specifically, treated mice saw…
Inhibiting a specific immune signaling pathway in the brain may reduce inflammation and seizure susceptibility, making it a potential new treatment target for Dravet syndrome, according to a study in mice. The study led by Li Gan, PhD, a professor at Weill Cornell Medical College in New York, found…
Patients will be randomly assigned to groups in a Phase 3 clinical trial testing bexicaserin for seizures associated with Dravet syndrome, according to the treatment’s developer. Lundbeck said it expects to complete patient randomization — assigning participants to groups receiving the treatment or a placebo — within…
Across a group of children with severe childhood epilepsies, including Dravet syndrome, genetic test results did not independently predict long-term seizure or developmental outcomes, according to a new study. “Although … genetic testing has diagnostic and precision-medicine selection utility in childhood-onset [developmental and epileptic encephalopathies], long-term seizure and developmental…
The first child has received ION337 in an open-label Phase 1/2 study, marking the start of clinical testing of this experimental therapy for Dravet syndrome. The study, called ASCEND (NCT07531745), is recruiting up to 32 children, ages 2 to 12, who have been diagnosed with Dravet syndrome and…
No new or unexpected safety issues were identified in a long-term study that tracked outcomes in people with Dravet syndrome taking the antiseizure medication Fintepla (fenfluramine). Of 265 people with Dravet syndrome, only about one in 10 experienced a side effect that investigators judged…
Enrollment has been completed in a Phase 3 clinical study of zorevunersen, an experimental treatment for Dravet syndrome being developed by Stoke Therapeutics, keeping the company on track to begin a rolling New Drug Application (NDA) submission with the U.S. Food and Drug Administration (FDA) in early…
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