News

For the first time, researchers have described a dysfunction in the brain’s GABAergic system that could increase the frequency and severity of epileptic seizures in patients with Dravet syndrome. The findings also highlight the therapeutic potential of cannabis derivatives, including cannabidiol, that are able to target GABAA receptors and…

Fenfluramine, originally developed as an appetite suppressant, shows potential as an anti-epileptic therapy to reduce the seizure rate in Dravet syndrome patients, a review study suggests. The study, “Individualized treatment approaches: Fenfluramine, a novel antiepileptic medication for the treatment of seizures in Dravet syndrome,” was published in…

The Department of Justice and the U.S. Drug Enforcement Administration (DEA) have cleared the use of the medicine Epidiolex by placing it in schedule V of the Controlled Substances Act (CSA). Schedule V is the least restrictive classification of the CSA, meaning that the agent is considered to…

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

A zebra fish model of Dravet syndrome may be one of the best animal models to identify new therapeutic candidates and achieve effective personalized medicine, according to a review study. The study, “Preclinical Animal Models for Dravet Syndrome: Seizure Phenotypes, Comorbidities and Drug Screening,” was published…

The U.S. Food and Drug Administration has approved Fycompa (perampanel) — alone or as an add-on — for the treatment of focal seizures, which affect only one part of the brain, with or without secondary generalized seizures, in children ages 4 and older. The approval includes Fycompa administered as a…

Stoke Therapeutics’ targeted-augmentation of nuclear gene output (TANGO) technology effectively increased the levels of the SCN1A gene and of its encoded protein in mice with Dravet syndrome. The company is currently conducting additional preclinical studies to evaluate the functional impact of this potential treatment strategy for Dravet syndrome. Isabel…

For the first time, positron emission tomography (PET) scans revealed a significant decrease in the amount of glucose uptake in the brains of children with Dravet syndrome. Interestingly, this reduction was more evident in older children. However, it is still unclear if the reduction in glucose uptake is a…

Supernus Pharmaceuticals now holds the worldwide rights — except in certain markets in Asia — to develop the investigational therapy BIS-001ER, renamed SPN-817, for Dravet syndrome, after a merger agreement to acquire Biscayne Neurotherapeutics, according to a press release. “We are excited to add…

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…