Marisa Wexler, MS,  senior science writer—

Ataluren, in development by PTC Therapeutics as a possible treatment for Dravet syndrome, showed an acceptable safety profile in a small clinical trial. That trial, conducted at a single site in New York City, involved 15 children with either Dravet syndrome due to mutations in the CDKL5…

Zogenix has launched a global access program for Fintepla (fenfluramine) to make the medication available to more patients with Dravet syndrome. In the United States and the European Union, Fintepla is approved as an add-on treatment to help prevent seizures in people with Dravet syndrome, ages…

Fintepla has no substantial impact on growth, in terms of height and weight, in children with Dravet syndrome, clinical trial data suggest. The findings were shared at the 2020 American Epilepsy Society (AES) Annual Meeting, in the presentation, “Treatment with Fintepla (fenluramine) in Patients with…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

Long-term treatment with Fintepla can reduce the frequency of seizures in children and adolescents with Dravet syndrome, according to two-year interim data from an open-label extension study. Study findings were presented by Zogenix, the therapy’s developer, in a poster titled “Long-Term (2-Year) Safety and Efficacy…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

Following consultation with the U.S. Food and Drug Administration (FDA), Eisai has initiated a Phase 3 clinical trial called MOMENTUM 1 to evaluate lorcaserin as a potential treatment for Dravet syndrome. Early data has suggested that lorcaserin may reduce the frequency of seizures among Dravet…

The United Kingdom Home Office has changed the classification of GW Pharmaceuticals‘ Epidyolex — a treatment for epileptic conditions including Dravet syndrome — from a Schedule 2 drug to a Schedule 5 drug. This move will make the medication easier to obtain. “The decision to move Epidyolex…

Stoke Therapeutics plans to begin enrollment later this year of Dravet syndrome patients for part A of its Phase 1/2a MONARCH trial to evaluate the investigational therapeutic STK-001, according to a press release. Most people with Dravet syndrome have mutations in one of the two copies (one…

Heart abnormalities are unlikely to be the reason behind the high rate of Sudden Unexpected Death in Epilepsy (SUDEP) in people with Dravet syndrome, a new study suggests, though further research is needed. The study, “Cardiac arrhythmias in Dravet syndrome: an observational multicenter study,” was…