A Prospective, Longitudinal, Observational Study in Individuals With Angelman Syndrome to Enable Endpoint Development for Interventional Clinical Trials (FREESIAS)
Angelman syndrome (AS) is a neurodevelopmental disorder, for which there are currently no established endpoints to holistically measure meaningful change in clinical trials. The FREESIAS study aimed to identify potential endpoints by following individuals with AS and a number of typically developing children for one year. A wide range of clinical outcome assessments, digital health technologies and overnight electroencephalography were tested across key AS characteristics, performed at home and in clinic.