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)…
News
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…
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
Activating the SCN1A gene, which is deficient in most patients with Dravet syndrome, reduced febrile seizures and improved behaviors in a mouse model, according to a recent study. The findings support a potential new approach to treat the cause of epilepsy…
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience. Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…
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