News

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…

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

Bringing together patients and their families — virtually, this year — the Dravet Syndrome Foundation (DSF) will present its fourth annual Day of Dravet workshop Oct. 17. The free event is an opportunity to learn more about treatment options and the latest Dravet syndrome research, connect…

The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), recommended lowering the age limit of the anti-seizure medication Fycompa (perampanel) to 4 for children with focal-onset seizures and to 7 for those with primary generalized tonic-clonic seizures…