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…
News
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…
News
Fintepla Reduces Seizure Frequency in Dravet Syndrome Youngsters, Pivotal Phase 3 Trial Reports
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,…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…
The Epilepsy Foundation’s Community Day, being held virtually this year, will offer patients, caregivers, and advocates the latest information about epilepsy and an opportunity to connect with others. “Innovation and Action to End Epilepsy,” a free national conference on Aug. 29, will focus on advocacy and a variety…
Recent Posts
- Cell study offers new clues to Dravet seizure triggers
- Epidiolex reduces seizures in rare Dravet syndrome cases: Study
- Faster-than-expected enrollment seen in trial testing zorevunersen for Dravet
- The little lost journal that brings back memories of our Dravet journey
- FDA grants breakthrough status to new gene therapy for Dravet syndrome
- First look at ETX101 suggests early benefits for children with Dravet
- Grief comes in waves and reminds me of the start of a long journey
- Stoke readies to meet with FDA on 4 years of zorevunersen study data
- Continuing family traditions helps keep my daughter’s memory alive
- FDA awards RMAT designation to Dravet syndrome gene therapy