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The National Institute of Health and Care Excellence (NICE), an arm of U.K.’s public health system, favors use of the oral cannabidiol solution Epidyolex as an add-on to clobazam to treat seizures associated with Dravet syndrome and Lennox Gastaut syndrome (LGS) in patients in England starting…

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

Treatment with Zogenix‘s Fintepla (ZX008) provides a sustained and meaningful reduction in convulsive seizure frequency in young children with Dravet syndrome, with similar clinical efficacy to that seen in older patient groups, according to results from a long-term open-label extension (OLE) study. Fintepla’s use also holds the potential…

Managing Dravet syndrome requires a multidisciplinary approach open to both pharmacological and non-pharmacological treatments, because of the severe nature of this disease and its poor prognosis, a review study reports. The study, “Dravet Syndrome: An Overview,” was published in Cureus. Dravet syndrome is a severe type of…

Neucyte and Trillium Therapeutics have established a license agreement to develop a new therapy for drug-resistant epilepsies, including Dravet syndrome and related disorders. The agent, whose name is still undisclosed, has shown promising results during studies carried out by the National Institute of Neurological Disorders and…

Ovid Therapeutics‘ investigational treatment soticlestat (OV935/5/TAK935) was found to progressively reduce seizure frequency — by up to 90% — in adults with rare and hard-to-treat epilepsies, including Dravet syndrome, after one year of treatment, according to preliminary data from the ENDYMION study. Topline results from the…

Seizures in people with Dravet syndrome may be associated with abnormal serotonin levels and activity in the brain, according to a mouse study. These results shed light on the potential of serotonin-boosting therapies, such as Fintepla (ZX008), to reduce seizures in Dravet patients. The study, “…

Zygel (ZYN002) — a cannabis-based gel formulation developed by Zynerba Pharmaceuticals — can significantly reduce seizures by up to six months in children and adolescents with developmental and epileptic encephalopathies, according to Phase 2 trial data. Developmental and epileptic encephalopathies, or DEE, is an umbrella term that…

Selectively blocking NaV1.6 — a sodium channel known to be overactive in some epileptic conditions — may offer a way of treating people with Dravet syndrome, according to work in a zebrafish model. The study, “NaV1.1 and NaV1.6 selective compounds reduce the behavior phenotype in a…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…