New hope for kids with devastating genetic epilepsy

This early-stage study is testing if a medication called glycerol phenylbutyrate is safe and well-tolerated in children with rare genetic disorders that cause severe epilepsy and developmental delays. The study will enroll up to 50 children, from infants to teenagers, who have conditions like STXBP1 or SLC6A1 disorders. The main goal is to check for side effects and see if children can take the medication regularly, while also measuring drug levels in the blood.