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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…

Confirmation of Dravet syndrome by genetic analysis and identification of gene mutations is important to provide patients with adequate treatment and follow-up, according to a case report. The report, “Dravet Syndrome in Lebanon: First Report on Cases with SCN1A Mutations,” was published in Case Reports in Medicine.

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bathdissertation, she analyzed Brexit’s long-term impact…

Multimodal analysis that incorporates data obtained from different methodologies seems to be the best approach to build a solid classification system for SCN1A genetic variants associated with Dravet syndrome, a study says. The study, “Multimodal analysis of SCN1A missense variants improves interpretation of clinically relevant variants in…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…