Author Archives: Joana Carvalho

Jazz Pharmaceuticals is set to acquire GW Pharmaceuticals and gain access to a diversified pipeline of cannabis-based medications, including Epidiolex (cannabidiol), which is approved to treat seizures in patients with therapy-resistant forms of epilepsy, such as Dravet syndrome. The transaction, which involves a cash-and-stock deal totaling…

Diacomit (stiripentol) is safe and effective at reducing the frequency of different types of seizures in people with Dravet syndrome when used routinely, including up to about two years, according to interim data from an ongoing study of its real-life use in Japan. Findings from the study, “…

By implementing a free-of-charge epilepsy genetic testing program, it is possible to lower the average age of molecular diagnosis of children with epileptic disorders caused by mutations in the SCN1A gene, such as Dravet syndrome, from more than 6 to less than 2 years of age. The study with…

Children with Dravet syndrome receiving Fintepla (fenfluramine) to control their seizures are unlikely to develop cardiac valvulopathy — a disease in which at least one of the four heart valves start to malfunction — or pulmonary arterial hypertension (PAH), according to interim data…

Used in addition to standard treatments, therapeutic hypothermia — based on lowering the body’s temperature — can shorten the duration of long-lasting seizures in drug-resistant forms of epilepsy, including Dravet syndrome, a study finds. The study, “Therapeutic hypothermia for pediatric refractory status epilepticus…

Epidiolex, the first plant-derived cannabidiol (CBD) medication to be approved by the U.S. Food and Drug Administration (FDA), can reduce premature mortality and alleviate several behavioral deficits in mouse models of Dravet syndrome, according to data from two studies. The results, “…

In addition to promoting the release of serotonin, a brain chemical typically associated with feelings of well-being and happiness, Fintepla (fenfluramine) also seems to control the activity of sigma-1 receptors found on the surface of nerve cells, a study has found. This dual mechanism of action is…

An antisense oligonucleotide therapy that lowers the amount of Scn8a transcripts — RNA molecules used as a template for the production of proteins — delayed seizure onset and prolonged the lifespan of mice in a model of Dravet syndrome and SCN8A encephalopathy, a study found. The study, “…

Fintepla (fenfluramine) reduced the frequency of convulsive seizures in children and adolescents with Dravet syndrome who responded poorly to treatment regimens that included Diacomit (stiripentol), Phase 3 trial results show. The findings were reported in a study, “Fenfluramine for Treatment-Resistant Seizures in…

STK-001, Stoke Therapeutics’s investigational therapy for Dravet syndrome, prolongs survival and reduces the frequency of seizures in a mouse model of the disease, according to preclinical results. Findings from these studies were presented by the company in a poster, titled “Targeted Augmentation of Nuclear Gene Output…