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Dravet Syndrome

Old Weight-Loss Drug Cuts Seizures in Dravet Syndrome

A drug that has previously been used as a weight-loss agent dramatically reduces seizures in young patients with Dravet syndrome (DS), new research shows.   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 Leuv...

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.

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, o...

Interview With A Neurologist: UNDERSTANDING CAUSES OF SUDEP

What is sudden unexpected death in epilepsy (SUDEP)? Currently, the accepted definition of SUDEP is the sudden unexpected witnessed or unwitnessed, non-traumatic and non-drowning death in people with epilepsy, with or without evidence of a seizure. It doesn’t necessarily have to be in the known context of a seizure, excluding documented status epilepticus, where people have seizure after seizure. Typically, what happens is that someone who’s known to have epilepsy is found to have died without any obvious explanation as to why. Even a post-mortem examination doesn’t reveal a structural or toxicological cause of death. It may or may not be known whether a seizure had occurred and no other cause of death has been identified.

Reduction of “Tau” Protein in the Brain Blocks Development of Dravet Syndrome

In this article posted on Medical Express, research is presented that supports a theory that reducing brain levels of tau protein can effectively block the development of Dravet Syndrome in lab-mouse studies. The treatment not only suppressed seizure activity and prolonged life, but also improved abnormal behavior and cognition that can accompany the syndrome. Excerpt: Previous studies from this group have shown that lowering tau levels reduces abnormal brain activity in models of Alzheimer’s disease, but this is the first demonstration that tau reduction may also be beneficial in intractable genetic epilepsy. “It would really be wonderful if tau reduction turned out to be useful not only in Alzheimer’s disease, but also in other disabling neurological conditions for whic...

Potential Cure for Epilepsy: Cannabis Vastly Reduces Seizures in Boy with Dravet Syndrome

Scientists are looking at the potential use of cannabis to treat and potentially cure severe forms of epilepsy. The medical benefits of medical marijuana for neurological conditions like epilepsy are highly debated. Examining the potential use of the drug, a series of articles in the journal Epilepsia, a journal of the International League Against Epilepsy, looks at its potential use for treating the syndrome. Edward Maa, from the Comprehensive Epilepsy Program at Denver Health in Denver, Colorado, looks at a case study of a child with Dravet syndrome – a very severe form of epilepsy. In the case, a mother provided her child with a strain of medical marijuana high in Cannabidiol (CBD) and tetrahydrocannabinol (THC) known as Charlotte’s Web. When used with the child’s normal dru...

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