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People with different types of epilepsy have significant brain structure abnormalities, including alterations in the thickness and volume of the gray matter of several regions of the brain, according to a large-scale analysis. Scientists in the international research consortium ENIGMA performed the largest neuroimaging analysis of epilepsy to date…

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…

Two teenage girls with Dravet syndrome became seizure-free after starting contraceptive hormonal therapies containing synthetic estrogen and progesterone, according to a recent case report. The girls had begun taking a birth control pill (patient 1) or received a contraceptive implant (patient 2) at the ages of…

Current first-line therapies for Dravet syndrome have limited effectiveness, but treatment candidates now undergoing clinical testing show significant promise, researchers suggest in a recent review. The study, “Treatment Strategies for Dravet Syndrome,” was published in the journal CNS Drugs. Accurate diagnosis of Dravet syndrome is possible within the…

Biscayne Neurotherapeutics has licensed the Chinese rights to its investigative antiepileptic therapy BIS-001ER to Global Drug Development Centre (GDCC) China in Taiwan, China, Hong Kong, and Macau. Biscayne is developing BIS-001ER to treat refractory forms of focal epilepsy, including catastrophic pediatric-onset epilepsies such as Dravet syndrome and Lennox Gastaut. “BIS-001ER is…

Raising a child with Dravet syndrome is hard enough without the added stress of fighting with insurance companies to cover the expensive medications such kids need to stay alive. Beth Fox ought to know. Her 9-year-old daughter, Brenna, has Dravet — a rare, catastrophic, lifelong form of drug-resistant epilepsy that…

An RNA splicing technique that Stoke Therapeutics is developing led to the production of a protein that’s lacking in Dravet syndrome, a study in mice showed. Stoke discussed the approach at the Oligonucleotide & Peptide Therapeutics Conference in Boston, March 26-28. Dr. Charles R. Allerson, Stoke’s vice president of chemistry, delivered the…

Noninherited genetic mutations affecting the SCN1A gene may help explain almost 1% of Dravet syndrome cases of unknown cause, according to researchers from Japan. Genetic disorders are commonly inherited from parents who are carriers of the abnormal gene variant that cause the disease. If the disease is recessive, it means the…

Epidiolex, GW Pharmaceuticals’ investigational cannabis-based oral therapy, was proven safe in a Phase 2 clinical trial testing it in young children with Dravet syndrome. The therapy is a plant-derived cannabidiol being developed for patients with treatment-resistant forms of epilepsy including…

GW Pharmaceuticals announced that it recently received allowance notices from the U.S. Patent and Trademark Office (USPTO) for five new patent applications for Epidiolex (cannabidiol). Three of these applications specifically focus on methods to reduce the frequency of seizures in patients with a form of Dravet syndrome. Notices of allowance…