News

June is Dravet Syndrome Awareness Month, and advocacy groups in the U.S. and U.K. are launching initiatives to raise awareness of Dravet syndrome and raise funds to fuel research and support families affected by this condition. In the U.S., this month of awareness is being…

A one-time treatment with Encoded Therapeutics’ experimental gene therapy ETX101 was generally well tolerated and associated with seizure reductions for up to one year in children with Dravet syndrome, a severe form of epilepsy that begins in infancy. That’s according to new data from the first phase of…

Zorevunersen, Stoke Therapeutics’ investigational therapy for Dravet syndrome, remained generally well tolerated and was associated with sustained seizure reductions and cognitive and behavioral improvements for up to four years. The findings come from two long-term extension studies — SWALLOWTAIL (NCT04740476) and LONGWING (ISRCTN12811235) — that followed participants…

Encoded Therapeutics announced that the first participant has been dosed in a pivotal clinical trial testing ETX101, the company’s experimental one-time gene therapy for Dravet syndrome, which is recruiting children and adolescents with this severe form of epilepsy. The pivotal study is the second part of the…

Diseases that mimic Dravet syndrome symptoms are usually caused by mutations in genes other than SCN1A — the gene most often mutated in Dravet — and identifying the exact genetic cause, through testing, is key to guiding appropriate treatment strategies, according to a new study from China. While most…

Beyond reducing seizures, add-on treatment with Fintepla (fenfluramine) may improve daily functioning in areas such as communication, attention, and awareness of surroundings in adults and children with Dravet syndrome, especially younger patients and those who engage more frequently in rehabilitation, an Italian study found. “Caregivers noticed meaningful improvements in…

Caregivers of people with Dravet syndrome frequently experience significant mental health challenges, including anxiety and depression, sleep problems, fatigue, stress, and social isolation, according to a systematic review of studies. The findings, published in the journal Epilepsy, highlight the emotional burden placed on caregivers and underscore the need for routine assessment…

Astrocytes change how they connect and function in a mouse model of Dravet syndrome — even without visible brain damage — which likely worsens seizures and cognitive problems, making them a potential target for future treatments, according to a study. These support cells in the brain help…

Dysfunction in mitochondria — the cell’s energy production centers — was frequently seen in children with developmental and epileptic encephalopathies (DEEs), including Dravet syndrome, even when mitochondrial disease was not the underlying cause, a new study found. Many children with these genetic conditions, which are marked by early-onset seizures…

Encoded Therapeutics has met with the U.S. Food and Drug Administration (FDA) and the two have aligned on the design of a pivotal clinical trial to support a potential approval filing for ETX101, a one-time gene therapy designed to address the underlying cause of Dravet syndrome. ETX101 is…