Dravet syndrome is a severe type of childhood epilepsy characterized by prolonged seizures that begin in the first year of life. It is a rare condition that affects one in 20,000 to 40,000 people worldwide. (more…)
Cannabidiol is an effective and generally well-tolerated add-on treatment for drop seizures in patients with Lennox-Gastaut syndrome, according to research published in The Lancet.
“Patients with Lennox-Gastaut syndrome, a rare, severe form of epileptic encephalopathy, are frequently treatment resistant to available medications,” Elizabeth A. Thiele, MD, from the pediatric epilepsy program at Massachusetts General Hospital, and colleagues wrote. “No controlled studies have investigated the use of cannabidiol for patients with seizures associated with Lennox-Gastaut syndrome.”
A new phase 3 study showed that compared with patients taking placebo, those taking low-dose fenfluramine (ZX008, Zogenix) experienced a statistically significant reduction in convulsive seizure frequency.
“The study showed a really significant and clinically meaningful decrease in seizure frequency. In about 70% of patients taking fenfluramine, the number of seizures per month went from about 40 to one or two per month,” lead author Lieven Lagae, MD, PhD, professor, University of Leuven, Belgium, head of the Pediatric Neurology Department, and director of the Childhood Epilepsy Program at the University of Leuven Hospitals, told Medscape Medical News.
The findings were presented here at the American Epilepsy Society (AES) 71st Annual Meeting 2017. (more…)
High Seizure Frequency in Children with Dravet Syndrome Negatively Impacts Quality of Life, New International Caregiver Survey Finds
Global survey of 584 caregivers of children, young adults and adults with Dravet illustrates the detrimental effect of debilitating, frequent seizures on quality of life and co-morbidities: Results suggest a need for more effective antiepileptic treatments.
The Dravet Syndrome European Federation and Zogenix, Inc. (NASDAQ:ZGNX), a pharmaceutical company developing therapies for the treatment of rare central nervous system (CNS) disorders, announced today the publication of the initial results from a landmark survey into the clinical, social and economic consequences of Dravet syndrome on children, young adults and their families in Developmental Medicine & Child Neurology. (more…)
Orphan Drug Designation From the U.S. FDA for EPX-100 and EPX-200 in the Treatment of Patients With Dravet Syndrome
The U.S. Food and Drug Administration has granted Orphan Drug Designation to EPX-100 and EPX-200 for the treatment of patients with Dravet syndrome. Dravet Syndrome qualifies as a rare pediatric disease under Section 529 of Food, Drug, and Cosmetic Act.
The rare, catastrophic, lifelong form of epilepsy begins in the first year of life with frequent or prolonged seizures. Intellectual disability, behavioral abnormalities, gait and motor dysfunction, and increased mortality are commonly observed as the disease progresses. Patients with Dravet Syndrome also suffer with life-threatening seizures that cannot be adequately controlled by available medications, and face a 15-20 percent mortality rate due to SUDEP (Sudden Unexplained Death in Epilepsy), seizure-related accidents such as drowning, or infections. In most cases, the disease is caused by heterozygous de novo mutations or gene deletions of SCN1A, a gene encoding a brain voltage-gated sodium channel (Nav1.1). (more…)