News

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…

GW Pharmaceuticals‘ Epidiolex, the first plant-derived cannabidiol (CBD) medication to be approved by the U.S. Food and Drug Administration (FDA), reduces the frequency of epileptic seizures when used as an add-on therapy in children with Dravet syndrome, a Phase 3 clinical trial shows.

Sudden unexpected death in epilepsy (SUDEP) occurs more frequently during early evening and is significantly prevented by prolonged use of the ketogenic diet, research in a mouse model of Dravet syndrome (DS) suggests. The reasons why this happens are unclear, and should be examined in more depth by future studies, but…

Compared with its competitors, Diacomit (stiripentol) remains in the top position as an add-on therapy to control epileptic seizures in young children with Dravet syndrome, or in those who are affected by repeated and prolonged seizures, according to an expert review study. The study, “Stiripentol for the…

The U.S. Food and Drug Administration (FDA) refused the New Drug Application (NDA) for Fintepla (ZX008), Zogenix‘s investigational anti-seizure therapy for patients with Dravet syndrome. According to statements from the FDA, the Refusal to File (RTF) letter was issued because the NDA submitted by Zogenix…

A wide variety of gait alterations have been reported for patients with Dravet syndrome, but the most common seems to be the crouch gait, a posture in which patients are unable to maintain an upright posture while walking, a review study has found. The study, “Gait deviations in…

Despite the promising anti-seizure effects of cannabinoids in children with Dravet syndrome and Lennox-Gastaut syndrome, more research is needed to understand how these compounds work and their potential benefits for other neurological disorders, according to a study. The study, “Safety, efficacy, and mechanisms of action of cannabinoids in…

In a departure from general government policy, Japan has approved the cannabis compound Epidiolex for clinical trials in epileptic patients with Dravet and Lennox-Gastaut syndromes. Although the country prohibits cannabis-based agents overall, tightly monitored hospital trials are exempt from its Cannabis Control Act. Epidiolex is a plant-derived…

Zogenix‘s investigational therapy Fintepla (ZX008) to treat epileptic seizures associated with Dravet syndrome will be marketed in Japan by pharmaceutical company Nippon Shinyaku. Fintepla is a low-dose oral solution of fenfluramine hydrochloride, a compound derived from the stimulant amphetamine. Patients take it with other epileptic treatments…

The impact of Dravet syndrome on the quality of life of patients and caregivers is generalizable across Western countries, and should be targeted as part of new therapy evaluations, research suggests. The findings were published in an article, “Perception of impact of Dravet syndrome on children…