News

New insights into what causes small brain vessels to leak and promotes blood-brain barrier dysfunction in people with epilepsy were revealed in a recent mouse study. Findings were published in the study, “Matrix Metalloproteinase-Mediated Blood-Brain Barrier Dysfunction in Epilepsy,” in the Journal of Neuroscience.

The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

A combination of high body temperature, seizures, and SCN1A mutations can cause the rapid accumulation of fluid in the brain and cardiac arrest, potentially leading to sudden death in Dravet syndrome patients, according to a case report. The study, “Can…

Children with Dravet syndrome are at risk of coma after prolonged epileptic seizures, especially when associated with high fever, according to a large Chinese retrospective study. The study, “The clinical outcome and neuroimaging of acute encephalopathy after status epilepticus in Dravet syndrome,” was published in the journal…

An approved cancer medication was able to reduce average daily seizures by 60 percent in zebrafish and mice with epilepsy. That finding was in an article, “A novel metabolism-based phenotypic drug discovery platform in zebrafish uncovers HDACs 1 and 3 as a potential combined anti-seizure drug target,” that was…

Inhibiting the communication between nerve cells located on different sides of the brain can prevent epilepsy seizures from spreading, a mouse study shows. The study “Dynamic interaction of local and transhemispheric networks is necessary for progressive intensification of hippocampal seizures” was published in the journal Scientific…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

An investigative oral medication called ZX008 was seen to effectively reduce the frequency of seizures in children and adolescents with Dravet syndrome, including those who had not responded to prior therapies, and may come to offer  “a new treatment option.” These findings were given in two scientific posters during the…

An advisory committee of the U.S. Food and Drug Administration has recommended approval of Epidiolex, an oral cannabidiol medicine, to treat two rare forms of severe epilepsy. The unanimous April 19 vote is non-binding, but makes it more likely that the FDA will decide on June 27 to give…