News

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the U.S. sponsor for Rare Disease Day on Feb. 28. The annual awareness day spotlights approximately 7,000…

Single and multiple doses of Stoke Therapeutics’ investigational therapy STK-001 were well-tolerated and led to a significant reduction in the frequency of convulsive seizures among children and adolescents with Dravet syndrome in the first three months of follow-up, interim data from the ongoing Phase 1/2a MONARCH trial show.

ReS3-T, an investigational medicine developed by reMynd for therapy-resistant epilepsies such as Dravet syndrome, has been accepted into the National Institute of Neurological Disorders and Stroke (NINDS) Epilepsy Therapy Screening Program (ETSP). The ETSP identifies potential therapies for drug-resistant epilepsy and disease prevention and modification by allowing researchers…

Virpax Pharmaceuticals has acquired exclusive worldwide rights from Nanomerics to develop a nasal spray of cannabidiol to treat epilepsy in adults and children, including those with Dravet syndrome. Under the agreement, Virpax can develop, manufacture, and sell VRP324, an investigational formulation of cannabidiol (CBD) administered using a nasal…

Stoke Therapeutics’ investigational therapy STK-001 is generally safe and results in a trend toward a reduction in seizure frequency in children and adolescents with Dravet syndrome, according to interim data from the ongoing Phase 1/2a MONARCH trial. These promising early findings, concerning STK-001’s single doses up to 30…

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…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Neuroene Therapeutics‘ NT102, a potential anti-seizure therapy for Dravet syndrome. NT102, given orally, has been shown to prevent seizures in several animal models, with “excellent protection” against such attacks in Dravet syndrome, according to Neuroene. Orphan drug…

In the two years since the Epilepsy Foundation launched its nationwide Seizure Safe Schools initiative, a dozen states have enacted legislation aimed at providing a safe school environment for the approximately 470,000 children and teens in the U.S. who have an epileptic condition, including Dravet syndrome. The initiative…

Sporting purple gear and sharing personal stories, supporters are observing National Epilepsy Awareness Month in November to call attention to epilepsy and associated disorders, such as Dravet and Lennox-Gastaut syndromes. More than 65 million individuals globally live with the central nervous system disorder. One-third of those experience…

In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…