ResearchCLINICALTRIALSMay 7
Trial Completed: Gait in Rare Diseases (NCT05161494)
Researchers completed a study looking at whether treatments that help with walking problems in one rare genetic disorder (Dravet Syndrome) could also help people with two other rare genetic disorders: Tuberous Sclerosis Complex and STXBP1 Encephalopathy. The study enrolled 41 people total and aimed to see if the same approach to treating movement and walking difficulties caused by epilepsy and developmental delays could work across different genetic conditions.
WHY IT MATTERSIf successful, this research could lead to new treatment strategies for gait and movement problems in Tuberous Sclerosis Complex and STXBP1 Encephalopathy—conditions where walking difficulties significantly impact quality of life but have limited treatment options.