News

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,”…

Long-term treatment with Fintepla can reduce the frequency of seizures in children and adolescents with Dravet syndrome, according to two-year interim data from an open-label extension study. Study findings were presented by Zogenix, the therapy’s developer, in a poster titled “Long-Term (2-Year) Safety and Efficacy…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

The first patient has been enrolled in Epygenix Therapeutics’ multicenter Phase 2 clinical trial that will assess the effectiveness and safety of EPX-100 as an add-on therapy in children with Dravet syndrome whose…

Following consultation with the U.S. Food and Drug Administration (FDA), Eisai has initiated a Phase 3 clinical trial called MOMENTUM 1 to evaluate lorcaserin as a potential treatment for Dravet syndrome. Early data has suggested that lorcaserin may reduce the frequency of seizures among Dravet…