Marisa Wexler, MS,  senior science writer—

Japan’s Ministry of Health, Labour and Welfare has approved oral Fintepla (fenfluramine) as an add-on treatment for seizures associated with Dravet syndrome in adults and children 2 and older. The therapy is specifically indicated for patients whose seizures are not adequately controlled by other anti-seizure medications. The approval…

Treatment with soticlestat significantly reduced seizure frequency for children with Dravet syndrome or Lennox-Gastaut syndrome (LGS), another childhood disorder that causes seizures, in a Phase 2 clinical trial called ELEKTRA. Trial results were in the study, “A phase 2, randomized, double-blind, placebo-controlled study to evaluate the…

Adults with Dravet syndrome commonly experience walking difficulties that tend to worsen with age, a small study reports. “In this study, it was observed that gait parameters and parkinsonian symptoms were abnormal across all ages, but clearly worse in older patients, suggesting a progressive gait disorder,” researchers wrote. By…

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Eight adults with lifelong histories of epilepsy and developmental delays without any known cause were diagnosed with Dravet syndrome after undergoing genetic testing, a new study reports. The finding suggests “that a number of adult patients are currently undiagnosed and have unmet health needs,” according to researchers. “In our…

Vaccines for COVID-19 are generally safe for children with Dravet syndrome, and are unlikely to cause an increase in the frequency of seizures, whereas COVID-19-like disease often increases seizure frequency, according to a pair of surveys done in the U.K. Since the start of the pandemic, researchers have…

Treatment with STK-001, Stoke Therapeutics‘ investigational therapy for Dravet syndrome, has so far raised no safety concerns among children taking part in the ongoing Phase 1/2a MONARCH clinical trial, according to the results of a planned interim analysis. Early data also hint that the medication may reduce seizure…

Longboard Pharmaceuticals is planning to launch a Phase 1b/2a clinical trial early next year that will evaluate the company’s investigational therapy LP352 in adults with Dravet syndrome and other types of developmental and epileptic encephalopathies (DEEs). The trial will test the therapy’s effectiveness in lessening seizures in patients…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Ataluren, in development by PTC Therapeutics as a possible treatment for Dravet syndrome, showed an acceptable safety profile in a small clinical trial. That trial, conducted at a single site in New York City, involved 15 children with either Dravet syndrome due to mutations in the CDKL5…