News

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…

Vaccines against COVID-19 appear to be safe and well-tolerated in patients with Dravet syndrome, a U.K. survey indicates. A brief report on the survey’s top results was announced recently by Dravet Syndrome UK. The COVID-19 pandemic has had an incalculable impact across the world. Recently, several…

Takeda Pharmaceuticals has acquired the global rights to develop and market the investigational oral therapy soticlestat (OV935/TAK-935) for developmental and epileptic encephalopathies, including Dravet syndrome, the company has announced. Soticlestat was originally developed by Takeda and Ovid Therapeutics, after the companies entered into a collaboration in 2017.

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

In its 13th year, the nonprofit Dravet Syndrome UK (DSUK) — which seeks to improve the lives of those affected by the epilepsy disorder — has introduced its first strategic plan for supporting patients and their families through research, awareness, and education. The five-year strategy outlines how the DSUK…

The Dravet Syndrome Foundation (DSF) is promoting awareness of the new global health statistics codes now in effect for Dravet syndrome. Known as “ICD-10” codes, the designations could bring about improved patient outcomes and scientific knowledge of this severe type of epilepsy. The DSF is encouraging those…

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…