Problems with sleep and body temperature regulation in Dravet syndrome are linked through the hypothalamus — the part of the brain that regulates both — and targeting it may help develop better treatments, according to a study in a mouse model of the disease. “These findings highlight a connection between…
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Parents reported that their children with Dravet syndrome experienced more frequent, longer, and more intense seizures during heatwaves, according to a qualitative interview study. Parents described heatwaves, sudden temperature changes, water exposure, and even excitement during hot weather as common seizure triggers. Many said they reorganized daily routines and…
Despite treatment with available antiseizure medications, people with Dravet syndrome often still experience seizures, as well as nonseizure symptoms, a new study highlights. The study also found that many Dravet patients end up discontinuing or switching seizure medications due to side effects. Overall, these data “suggest that current treatments…
Behavioral and social difficulties are common in young children with Dravet syndrome, often emerging in toddlerhood and worsening through early childhood, according to a natural history study. The ENVISION (NCT04537832) study followed 58 children with Dravet syndrome, aged 6 months to 5 years, for up to two years.
An online occupational therapy program that coaches parents on how to help their children problem-solve may help young people with Dravet syndrome improve at daily tasks, a small study suggested. The program is based on the Cognitive Orientation to daily Occupational Performance (CO-OP) Approach, a therapy usually delivered directly…
Lab-grown nerve cells from people with Dravet syndrome have abnormal levels of chemical elements that regulate nerve cell function, including potassium, copper, and zinc, a study found. Researchers said these imbalances may contribute to abnormal brain electrical activity, which makes people with Dravet prone…
Epidiolex (cannabidiol) reduced seizure frequency in five children with Dravet syndrome caused by 2q24.3 microdeletions, according to a case series. Dravet syndrome is a severe form of epilepsy that begins in early infancy. A 2q24.3 microdeletion involves the loss of a segment of chromosome 2 that includes the…
Enrollment in a large clinical trial evaluating zorevunersen for Dravet syndrome is now expected to be completed in the second quarter of this year, earlier than previously anticipated, positioning the therapy candidate for a potential new drug application (NDA) submission in 2027. That’s according to Stoke Therapeutics, which is…
ETX101, an experimental one-time gene therapy for Dravet syndrome, has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA). The FDA grants this designation to investigational treatments for serious conditions that, based on early evidence, may offer a meaningful improvement over existing options. The designation…
ETX101, an investigational one-time gene therapy being developed by Encoded Therapeutics for children with Dravet syndrome, has shown a favorable safety profile and early signs of benefit in an ongoing clinical program. Called the POLARIS program, the clinical effort includes three Phase 1/2 trials of ETX101: ENDEAVOR (NCT05419492)…
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