Alice Melao,  —

Treatment with Zogenix‘s Fintepla (ZX008) provides a sustained and meaningful reduction in convulsive seizure frequency in young children with Dravet syndrome, with similar clinical efficacy to that seen in older patient groups, according to results from a long-term open-label extension (OLE) study. Fintepla’s use also holds the potential…

Zygel (ZYN002) — a cannabis-based gel formulation developed by Zynerba Pharmaceuticals — can significantly reduce seizures by up to six months in children and adolescents with developmental and epileptic encephalopathies, according to Phase 2 trial data. Developmental and epileptic encephalopathies, or DEE, is an umbrella term that…

Planning for a Phase 1 clinical trial to explore the safety and tolerability of escalating doses of EPX-100, a potential oral treatment for Dravet syndrome, is underway, Epygenix Therapeutics, the investigative therapy’s developer, announced. After completion of this first study in healthy individuals, the company plans to initiate…

Severe epilepsy in children with Dravet and Lennox-Gastaut syndrome can be safely treated with a medicinal cannabis oil that includes, at low dose, the intoxicating compound in marijuana known as THC, preliminary results of a Phase 1 clinical study suggest. “What makes these results really exciting is it opens up as…

Inhibition of an enzyme in the “powerhouses” of cells from the hippocampus can prevent seizures in mice with intractable Dravet syndrome, a study found. This discovery may pave the way for the development of therapies that could effectively reduce brain cells’ hyperactivity, known to increase susceptibility to seizures and…

Dravet syndrome is associated with a high prevalence of behavioral problems that can severely affect quality of life, compared with the general population or to patients with epilepsy but without this disease, a study reveals. These findings highlight the need for active management and treatment strategies to address such…

A single dose of Stoke Therapeutics’ antisense oligonucleotide, targeting the SCN1A gene, largely eliminated the incidence of seizures as well as sudden unexplained death in epilepsy (SUDEP) cases of mice with Dravet syndrome, a study showed. The most recent preclinical data on Stoke’s targeted augmentation of nuclear gene…

Nighttime sleep quality is poorer in Dravet syndrome patients and daytime sleepiness more pressing than patients with other types of epilepsy, an exploratory study reports. The problems are shared between both these patient groups, however. The study, “More daytime sleepiness and worse quality…

The Department of Justice and the U.S. Drug Enforcement Administration (DEA) have cleared the use of the medicine Epidiolex by placing it in schedule V of the Controlled Substances Act (CSA). Schedule V is the least restrictive classification of the CSA, meaning that the agent is considered to…

Stoke Therapeutics’ targeted-augmentation of nuclear gene output (TANGO) technology effectively increased the levels of the SCN1A gene and of its encoded protein in mice with Dravet syndrome. The company is currently conducting additional preclinical studies to evaluate the functional impact of this potential treatment strategy for Dravet syndrome. Isabel…