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…