News

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

Zogenix‘s investigational compound Fintepla (fenfluramine HCl oral solution) improves behavioral and emotional control in children and young adults with Dravet syndrome, according to recent data presented at the 72nd American Epilepsy Society Annual Meeting in New Orleans. These findings were based on a post-hoc analysis performed on patients who participated…

Add-on oral treatment with Epidiolex (cannabidiol) significantly lowered the frequency of convulsive seizures in patients with Dravet syndrome, according to topline results of a Phase 3 clinical trial. The study (NCT02224703) was the second double-blind, placebo-controlled Phase 3 clinical trial of GW Pharmaceuticals’ Epidiolex in the treatment of…

Diacomit (stiripentol) add-on therapy is more effective in children with Dravet syndrome who have pathogenic (disease-causing) SCN1A mutations than in those with variants of unknown significance and benign SCN1A mutations, a study has found. The study, “Efficacy of Stiripentol in Dravet Syndrome with or without SCN1A Mutations,” were…

Researchers have reported a case of a child with Dravet syndrome who began to have seizures triggered by wiping during diaper changes and whose seizures worsened after a mild head injury. The case, “Perineal stimulation triggering seizures in a child with Dravet syndrome,” was published as a…

Epidiolex (cannabidiol) oral solution is now available in the United States as a treatment for seizures in patients age 2 or older who have Lennox-Gastaut syndrome or Dravet syndrome. Epidiolex is developed by GW Pharmaceuticals and will be marketed in the U.S. by its subsidiary, Greenwich Biosciences. The anti-epileptic therapy…

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…