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Treatment with stiripentol reduces seizures and can resolve epileptic episodes, retrospective analysis of a small study in Turkish Dravet syndrome children suggests. The study, “Efficacy of Stiripentol and the Clinical Outcome in Dravet Syndrome,” was published in the Journal of Child Neurology. Stiripentol, sold as Diacomit by…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…

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…

Researchers have identified two simple diagnostic criteria to help healthcare practitioners make an early and correct diagnosis of Dravet syndrome in children living in a resource-limited setting in Africa. The study, “Dravet syndrome in South African infants: Tools for an early diagnosis,” was published in…

For the first time, researchers have described a dysfunction in the brain’s GABAergic system that could increase the frequency and severity of epileptic seizures in patients with Dravet syndrome. The findings also highlight the therapeutic potential of cannabis derivatives, including cannabidiol, that are able to target GABAA receptors and…

Fenfluramine, originally developed as an appetite suppressant, shows potential as an anti-epileptic therapy to reduce the seizure rate in Dravet syndrome patients, a review study suggests. The study, “Individualized treatment approaches: Fenfluramine, a novel antiepileptic medication for the treatment of seizures in Dravet syndrome,” was published in…

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…

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

A zebra fish model of Dravet syndrome may be one of the best animal models to identify new therapeutic candidates and achieve effective personalized medicine, according to a review study. The study, “Preclinical Animal Models for Dravet Syndrome: Seizure Phenotypes, Comorbidities and Drug Screening,” was published…

The U.S. Food and Drug Administration has approved Fycompa (perampanel) — alone or as an add-on — for the treatment of focal seizures, which affect only one part of the brain, with or without secondary generalized seizures, in children ages 4 and older. The approval includes Fycompa administered as a…