News

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…

Fintepla Improves Behavioral and Emotional Control in Dravet Patients, Analysis of Phase 3 Trial Shows

Zogenix‘s investigational compound Fintepla (fenfluramine HCl oral solution) improves behavioral and emotional control in children and young adults with Dravet syndrome, according to recent data presented at the 72nd American Epilepsy Society Annual Meeting in New Orleans. These findings were based on a post-hoc analysis performed on patients who participated…

Diacomit Add-On Therapy More Effective in Children With Dravet Syndrome Who Carry Pathogenic SCN1A Mutations, Study Shows

Diacomit (stiripentol) add-on therapy is more effective in children with Dravet syndrome who have pathogenic (disease-causing) SCN1A mutations than in those with variants of unknown significance and benign SCN1A mutations, a study has found. The study, “Efficacy of Stiripentol in Dravet Syndrome with or without SCN1A Mutations,” were…