Vaccines for COVID-19 are generally safe for children with Dravet syndrome, and are unlikely to cause an increase in…
Articles by Marisa Wexler, MS
Treatment with STK-001, Stoke Therapeutics‘ investigational therapy for Dravet syndrome, has so far raised no safety concerns among…
Longboard Pharmaceuticals is planning to launch a Phase 1b/2a clinical trial early next year that will evaluate the company’s…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for…
Ataluren, in development by PTC Therapeutics as a possible treatment for Dravet syndrome, showed an acceptable safety profile…
Zogenix has launched a global access program for Fintepla (fenfluramine) to make the medication available to more patients…
Fintepla has no substantial impact on growth, in terms of height and weight, in children with Dravet syndrome,…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
Long-term treatment with Fintepla can reduce the frequency of seizures in children and adolescents with Dravet syndrome, according to…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…