Dravet Syndrome News and Research

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Overactive brain immune response linked to rare, severe genetic epilepsy

An overactive immune response in the brain may play a role in Dravet Syndrome, a rare and severe genetic epilepsy that typically begins in infancy, according to Weill Cornell Medicine researchers.

29 Jul 2026

Base editing approach repairs deadly Dravet syndrome mutation in mice

Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially deadly form of childhood epilepsy. After the edit, the mice have far fewer seizures and live much longer.

13 May 2026

University of Chicago and IDefine launch research program to develop Kleefstra syndrome treatment

The University of Chicago Department of Chemistry, in partnership with the advocacy organization IDefine – The Kleefstra Syndrome Foundation, is launching a targeted research program to develop a new potential therapeutic strategy for Kleefstra syndrome (KLEFS).

26 Mar 2026

Novel gene regulation therapy passes key clinical milestone

Groundbreaking Phase 1/2a clinical trials co-led by Linda Laux, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, show that the first gene regulation treatment for epilepsy is safe and well tolerated by patients with Dravet syndrome for whom antiseizure medications are not effective.

4 Mar 2026

Experimental therapy dramatically reduces seizures in children with Dravet syndrome

A new experimental treatment for children with a hard-to-treat form of epilepsy is safe and can reduce seizures dramatically, helping them lead much healthier and happier lives, the findings of a UCL and Great Ormond Street Hospital-led international clinical trial show.

4 Mar 2026

Medical cannabis evidence falls short for most conditions

Medical cannabis lacks adequate scientific backing for most of the conditions it is commonly used to treat, including chronic pain, anxiety and insomnia, according to a comprehensive review led by UCLA Health.

3 Dec 2025

New short film sheds light on the impact of climate change on brain health

A powerful new short film, from the FutureNeuro Research Ireland Centre and RCSI University of Medicine and Health Sciences sheds light on the urgent need to address the impact of climate change on brain health.

19 May 2025

New study links metabolic defects to Dravet syndrome

New research from the University of Colorado Anschutz Medical Campus is the first to identify defects related to metabolism (how the body processes and uses energy) in lymphoblast cell lines (LCLs) derived from blood samples of children with Dravet syndrome.

25 Mar 2025

Gene therapy breakthrough offers hope for severe Dravet syndrome cases

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, paving the way for future treatments.

18 Feb 2025

Gene therapy shows promise in treating SCN1B-linked epileptic encephalopathies

Dravet syndrome and other developmental epileptic encephalopathies are rare but devastating conditions that cause a host of symptoms in children, including seizures, intellectual disability, and even sudden death.

14 Feb 2025

Exploring the potential of ketogenic diets in managing epilepsy

The mechanisms of action, efficacy, and side effects of ketogenic diet (KD) variations in epileptic patients.

26 Apr 2024

Experts shed light on how autism affects the diagnosis, treatment of epilepsy and vice versa

Compared with the general population, epilepsy is more common in people with autism - and autism is more common in people with epilepsy. How can autism affect the diagnosis and treatment of epilepsy, and vice versa? Joy Mazur spoke to Dr. Colin Reilly and Dr. Stéphane Auvin.

17 Nov 2023

High-dose CBD fails to relieve pain in knee osteoarthritis patients

Cannabidiol (CBD) is marketed by some suppliers as a painkiller, e.g. for osteoarthritis of the knee. Animal experiments have shown that the substance, which is extracted from the hemp plant, has an anti-inflammatory and pain-relieving effect in arthritis.

14 Nov 2023