News

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…

Fintepla (fenfluramine) oral solution, developed by Zogenix, significantly reduced seizure frequency in children and teenagers with Dravet syndrome, according to top-line results from a Phase 3 clinical trial.  This trial (Study 3)…

Children with Dravet syndrome receiving Fintepla (fenfluramine) to control their seizures are unlikely to develop cardiac valvulopathy — a disease in which at least one of the four heart valves start to malfunction — or pulmonary arterial hypertension (PAH), according to interim data…

The investigational oral therapy Soticlestat (OV935/TAK-935), developed by Ovid Therapeutics and Takeda Pharmaceuticals, safely and effectively lowers seizure frequency in children with Dravet syndrome and Lennox‑Gastaut syndrome (LGS), according to top-line data from a Phase 2 clinical trial. “We are extremely encouraged by these results, which…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

The first patient has been dosed in Stoke Therapeutics‘ Phase 1/2a MONARCH trial, which is investigating the safety, tolerability, and efficacy of STK-001 as a treatment for children and adolescents with Dravet syndrome. Enrollment in the trial is ongoing. People with Dravet, a severe type of epilepsy,…