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…
Discussion
Eleven years ago, I brought new life into the world in one of my favorite months of the year, when things begin to warm and blossom. May 19, 2015, was one of the three best days of my life — the day my daughter Austen was born. The two other…
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…
Becoming educated and informed about Dravet syndrome is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnist’s words of wisdom to help you along on your Dravet syndrome journey.
There is no cure for Dravet syndrome yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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