Forest Ray PhD,  —

Fintepla (fenfluramine) successfully managed the severe and difficult-to-treat seizures of a 20-year-old woman with Dravet syndrome, demonstrating the medication’s potential in real-life for treating such challenging cases, according to researchers. “The profound and sustained response to [Fintepla] seen in this patient is similar to that observed in double-blind clinical…

Caregivers of children with Dravet syndrome who are taking Fintepla (fenfluramine) report sustained seizure reductions and quality-of-life improvements, among other benefits, according to a recent study. These findings were presented as a poster titled “Caregivers’ Perspectives on the Long-term Seizure- and Non-seizure-related Benefits of Fenfluramine…

The risk of severe COVID-19 outcomes does not appear to increase in people with Dravet syndrome, although they still may need additional medical attention, according to the results of a recent survey in the U.K. Results of the survey, “The impact of COVID‐19 in Dravet syndrome:…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

The U.S. Food and Drug Administration (FDA) will allow an additional higher dose of STK-001 to be added to the Phase 1/2a MONARCH trial, which is evaluating the potential therapy’s safety and tolerability in treating children and adolescents with Dravet syndrome. The FDA had previously placed higher…

The first patient has been successfully dosed in a Phase 2 clinical trial testing Epigenix Therapeutics’ EPX-100 (clemizole) as an add-on therapy for children with Dravet syndrome. “We are very excited to announce that [the] first patient has been successfully dosed in our trial. First patient dosing 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…

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…

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…

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…