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...
PARIS (Reuters) – An estimated 2,150 to 4,100 children in France suffered a major malformation in the womb between 1967 and 2016 after their mothers took a treatment against epilepsy and bipolar disorders known as valproate, France’s drug regulator said on Thursday. Valproate, which has been manufactured in France by Sanofi under the brand Depakine in the field of epilepsy and Depakote and Depamide in bipolar disorders, is also believed to cause slow neurological development.
Promising results from a large-scale, controlled, Phase 3 clinical study of epilepsy patients being treated with cannabidiol will be presented next week at the American Academy of Neurology’s Annual Meeting in Boston on April 25. GW Pharmaceuticals’ liquid oral formulation of cannabidiol (CBD), called Epidiolex, is one of 500 compounds found in cannabis. Unlike the well-known compound, tetrahydrocannabinol (THC), CBD does not produce a “high” as the psychoactive component is absent. Results from the randomized, double-blind, placebo-controlled study found that almost 40 percent of people with Lennox-Gastaut syndrome (LGS) had at least a 50 percent reduction in drop seizures, compared to 15 percent taking a placebo. LGS is a severe form of epilepsy that often results in impaired intellectua...
Consider two children who have childhood absence epilepsy (CAE), the most common form of pediatric epilepsy. They both take the same drug—one child sees an improvement in their seizures, but the other does not. A new study in the Annals of Neurology identified the genes that may underlie this difference in treatment outcomes, suggesting there may be potential for using a precision medicine approach to help predict which drugs will be most effective to help children with CAE. The study was funded by the National Institute of Neurological Disorders and Stroke (NINDS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), both part of the National Institutes of Health. “A better understanding of genetic factors underlying a disease and the way t...
Drug-induced hypersensitivity reactions (DIHRs) are serious and life threatening. A common example is the use of the antiepileptic drug carbamazepine, but the mechanisms that trigger it are unclear. Current scientific consensus holds that people who have a specific variation of the ‘human leukocyte antigen B’ (HLA-B) gene, which provides the code for making a protein that plays a critical role in the immune system, are more at risk of DIHR. However, the mechanism linking this gene to DIHR is currently unknown. As this specific variation, called HLA-B*15:02, is fairly common in people of South-East Asian descent, this is a serious problem for clinicians in the region.
An epilepsy patient’s emotional well-being may be negatively impacted when changes are made to their antiepileptic drug (AED) regimen. These are the findings from a study published online in the journal Epilepsy and Behavior. In order to understand how AED changes affect patient emotions, researchers asked members of an online epilepsy community to participate in an online survey which consisted of 31 questions that rated their feelings on a recent AED change. In addition to the survey results, comments from epilepsy-related online forums and social media websites where people expressed their experiences with AED changes were also analyzed (termed passive listening statements).
Prolonged epileptic seizures may cause serious problems that will continue for the rest of a patient’s life. As a result of a seizure, neural connections of the brain may be rewired in an incorrect way. This may result in seizures that are difficult to control with medication. Mechanisms underlying this phenomenon are not entirely known, which makes current therapies ineffective in some patients.
Neurological diseases are best studied using human neurons, and a longtime goal — once thought impossible — has been to grow such neurons in the lab. Now researchers have su cceeded, maintaining adult neurons in culture for months, an achievement that opens a way to better understand how epilepsy treatments affect the brain.
Researchers have identified a network of 320 genes in the brain that are linked to epilepsy — a finding that could help address genetic anomalies that lead to the condition. In about 25 percent of cases, epilepsy has a clear cause, such as a head trauma or stroke. But most cases are the result of a genetic anomaly, researchers said in the study “Rare And Common Epilepsies Converge On A Shared Gene Regulatory Network Providing Opportunities For Novel Antiepileptic Drug Discovery.” It was published in the journal Genome Biology.
A naturally occurring compound often used in traditional Chinese medicine offered “robust” protection against induced seizures in mouse models of epilepsy and may prove of benefit to people — especially children — with treatment-resistant forms of the disease, according to a study published in the journal Frontiers in Pharmacology. Huperzine A, found in the club moss Huperzia serrata, is particularly helpful against febrile seizures that often occur in those with Dravet syndrome, a severe form of childhood epilepsy, and in genetic epilepsy with febrile seizures plus (GEFS+).
A study published in Neurology suggests that methylphenidate (Ritalin) may help some cognitive deficits in patients with epilepsy. To compare the potential efficacy of immediate-release methylphenidate (Ritalin) vs. placebo in treating cognitive deficits in epilepsy, researchers conducted a double-blind, randomized, single-dose, 3-period crossover study (n=35). Patients with epilepsy and chronic cognitive complaints participated in three medication visits (approximately 1 week apart) where they were administered one of the blinded preparations (placebo, methylphenidate 10mg or 20mg).