Brain-like organoids, or “mini-brains,” that are specific to affected brain regions and derived from patients could act as models for studying the underlying molecular mechanisms of Dravet syndrome, a study shows. Researchers created patient-derived organoids of the forebrain, a region enriched with types of nerve cells called GABAergic neurons…
News
For this year’s International Epilepsy Day on Feb. 13, supporters joined together to eliminate myths and misconceptions about epilepsy. Organized by the International Bureau for Epilepsy (IBE) and the International League Against Epilepsy (ILAE), the event is held on the second Monday of each February in an…
Caregivers of children with Dravet syndrome report that their child’s use of Fintepla (fenfluramine) significantly reduces seizure burden and leads to a number of quality-of-life benefits for their children, themselves, and the rest of their family. That’s according to a recent study conducted in Europe, which involved interviews…
Fintepla (fenfluramine) is associated with dose-dependent, clinically meaningful improvements in everyday behavioral and emotional control, as well as the ability to adapt to new situations, in preschool-aged children with Dravet syndrome. These benefits weren’t always tied to a clinically meaningful reduction in the frequency of seizures, suggesting the…
Long-term use of the sodium channel blocker phenytoin led to reductions in seizure frequency for four male patients with Dravet syndrome, according to a study in Australia. Weaning off the medication caused seizures to worsen, and re-starting it lowered seizure frequency and improved alertness. The findings are in…
IAMA Therapeutics has signed a second agreement with PsychoGenics to test the effectiveness of new therapy candidates in Dravet syndrome animal models. The companies entered into a service agreement last year to identify the effectiveness of new drug candidates…
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…
The efficacy and safety of Epidiolex, an oral cannabidiol, in children and adults with Dravet syndrome and Lennox-Gastaut syndrome (LGS) were supported in a real-world setting, with benefits similar to those seen in previous clinical trials and expanded access programs (EAPs), a Spanish EAP study reported. EAPs are…
A novel gene therapy that works to increase the levels of an enzyme called p38-gamma extended survival and reduced seizure activity in a mouse model of Dravet syndrome, a new study reports. “We have tested this therapy extensively, and rigorous assessment by independent labs has confirmed our results,” said…
The Dravet Syndrome Foundation (DSF) has announced the award of $690,000 in total research funding for five projects that aim to improve care for people with Dravet syndrome. The one- to two-year research grants will provide each project funding in amounts ranging from $75,000 to $165,000. Two of…
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