Across a group of children with severe childhood epilepsies, including Dravet syndrome, genetic test results did not independently predict long-term seizure or developmental outcomes, according to a new study. “Although … genetic testing has diagnostic and precision-medicine selection utility in childhood-onset [developmental and epileptic encephalopathies], long-term seizure and developmental…
News
The first child has received ION337 in an open-label Phase 1/2 study, marking the start of clinical testing of this experimental therapy for Dravet syndrome. The study, called ASCEND (NCT07531745), is recruiting up to 32 children, ages 2 to 12, who have been diagnosed with Dravet syndrome and…
No new or unexpected safety issues were identified in a long-term study that tracked outcomes in people with Dravet syndrome taking the antiseizure medication Fintepla (fenfluramine). Of 265 people with Dravet syndrome, only about one in 10 experienced a side effect that investigators judged…
Enrollment has been completed in a Phase 3 clinical study of zorevunersen, an experimental treatment for Dravet syndrome being developed by Stoke Therapeutics, keeping the company on track to begin a rolling New Drug Application (NDA) submission with the U.S. Food and Drug Administration (FDA) in early…
Most children and adults living with Dravet syndrome in the U.S. who remain on Fintepla (fenfluramine) for at least six months have fewer seizures and fewer hospital and emergency visits during that time, a study finds.
Use of cannabidiol — a chemical from the hemp plant used for treating epilepsy — as an add-on treatment for individuals with developmental and epileptic encephalopathies (DEEs), including Dravet syndrome, leads to meaningful reductions in seizures for about half of patients, a new study has found. “Adjunctive pharmaceutical-grade cannabidiol…
Treatment with Fintepla (fenfluramine) reduced convulsive seizures by at least half in nearly 60% of people with Dravet syndrome in a small, real-world study in Japan, with many responding at low doses, data show. Among the patients who responded to Fintepla — approved in the Asian nation since…
Neurospheres, 3D clusters of early-stage neural cells derived from people with Dravet syndrome, showed patient-specific protein patterns that varied in association with disease severity in a recent study. Researchers broadly analyzed proteins in the neurospheres and found that, despite a shared molecular foundation, each patient’s cells exhibited distinct patterns…
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…
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