News

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

Researchers found that patients with Dravet syndrome are at higher risk of suffering sudden unexpected death in epilepsy (SUDEP) due to genetic mutations in the SCN1A gene that predispose them to heart arrhythmias. University of Michigan Medical School investigators now plan to look at the potential use of already approved…

Vagus nerve stimulation (VNS) therapy can lead to more than $77,000 in cost savings over five years for patients with drug-resistant epilepsy (DRE), compared to anti-epileptic treatments, according to new research. The study, “Expected Budget Impact and Health Outcomes of Expanded Use of Vagus Nerve Stimulation Therapy…

Zogenix’s lead investigational compound ZX008 provided significant and long-term seizure reduction in Dravet syndrome patients, according to new clinical trial data. The company also revealed data from multiple studies assessing the psychological and socioeconomic impact of epileptic encephalopathies, including Dravet syndrome, in the United States and Europe. These findings were presented in multiple studies during…

Early diagnosis followed by treatment with anti-epileptic medications reduced the frequency of seizures and behavioral problems in three children with both Dravet syndrome and autism spectrum disorder (ASD), a study reports. The study, “Epileptic Encephalopathy and Autism: A Complex Interplay,” was published in the Journal of Pediatric…

GW Pharmaceuticals has presented positive data on the safety and effectiveness of Epidiolex, recently approved by the U.S. Food and Drug Administration (FDA) to treat epileptic seizures in Dravet and Lennox-Gastaut syndromes, as well as the potential mechanisms behind its anti-epileptic properties. The data were presented at the 13th Annual…

A new system of vagus nerve stimulation (VNS) therapy was implanted in the first patient with drug-resistant epilepsy, as part of a global registry launched by LivaNova to evaluate the treatment in a real-world setting. The CORE-VNS registry (NCT03529045) plans to include up to 2,000 patients with drug-resistant epilepsy to be…

The U.S. Food and Drug Administration has accepted a new drug application (NDA) for UCB’s Nayzilam (midazolam) nasal spray for the acute treatment of seizures including seizure clusters and acute repetitive seizures. Acceptance of this NDA could result in Nayzilam’s approval in the U.S. in early 2019, potentially…

The U.S. Food and Drug Administration (FDA) has approved Biocodex’s Diacomit (stiripentol) as an add-on therapy for seizures associated with Dravet syndrome in patients 2 years and older who are undergoing treatment with Onfi (clobazam), an anticonvulsant therapy marketed by Lundbeck. Diacomit is used as an adjunctive (add-on) therapy for treating …

Long-term use of sodium channel blockers in the first five years of disease has been linked to poorer cognitive function in Dravet syndrome patients. The study, “Influence of contraindicated medication use on cognitive outcome in Dravet syndrome and age at first afebrile seizure as a clinical predictor in…