News

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Activities ranging from submitting names for a virtual remembrance wall to swapping out Facebook photo frames are on tap for Dravet Awareness Month, set aside each June to call attention to a severe type of epilepsy that affects an estimated 1 in 15,700 individuals in the U.S. Awareness and…

Ovid Therapeutics has closed an agreement under which Takeda Pharmaceuticals will secure global rights to develop and market the investigational oral therapy soticlestat (previously TAK-935/OV935) for two rare epilepsy syndromes, one of which is Dravet syndrome, the company announced. Takeda had announced the exclusive agreement…

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…

Researchers have developed a mathematical model to identify electrical signal patterns in the brain that predict epileptic seizures in advance, of benefit to people with seizure-related disorders such as Dravet syndrome, a study reports.  The study, “…

The first participant has been enrolled in Envision, a natural history study that will follow children with Dravet syndrome for more than two years as it seeks to shed light on how this severe type of epilepsy affects…

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

Caregivers of children with Dravet syndrome who are taking Fintepla (fenfluramine) report sustained seizure reductions and quality-of-life improvements, among other benefits, according to a recent study. These findings were presented as a poster titled “Caregivers’ Perspectives on the Long-term Seizure- and Non-seizure-related Benefits of Fenfluramine…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…