News

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

While Dravet syndrome patients don’t appear to be at increased risk of severe COVID-19 complications, more study is needed about potentially adverse effects of prolonged social distancing and other preventative measures, a Dravet Syndrome UK (DSUK) survey indicates. The report gives a view into the the…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

The first patient has been successfully dosed in a Phase 2 clinical trial testing Epigenix Therapeutics’ EPX-100 (clemizole) as an add-on therapy for children with Dravet syndrome. “We are very excited to announce that [the] first patient has been successfully dosed in our trial. First patient dosing is…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

Dravet syndrome now has its own global health statistics codes — known as “ICD-10” codes — that potentially could result in improved patient outcomes and enhanced clinical and scientific knowledge of the genetic disorder. The National Center for Health Statistics has designated what are known as International…

National Epilepsy Awareness Month is underway, with a variety of events and activities aimed at calling attention to diseases such as Dravet and Lennox-Gastaut syndromes, and to epilepsy in general. Patients, caregivers, and advocates around the U.S. will share educational information, host fundraisers, don purple gear,…

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…

Fintepla — low-dose oral fenfluramine — moved one step closer to European approval, following a favorable opinion on its use as an add-on treatment for seizures associated with Dravet syndrome in patients ages 2 and older from the Committee for Medicinal Products for Human Use (CHMP), an arm…

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…