News

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…

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…