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Cannabidiol for severe behavioral manifestations in TSC, MPS III, and FXS794Trial registration: EudraCT: 2021-003250-23, registered 25 August 2022, https://www.clinicaltrialsregister.eu/ctr-search/trial/2021-003250-23/NL.Highlights of the study protocoly Addressing unmet patient needs for treatment of severe behavioral manifestations of rare genetic disorders. y Evaluation of a novel drug recently approved for certain rare epilepsy syndromes many of which characterized by behavioral problems. y Innovative trial design (series of N-of-1 trials) in rare disorders, applying evidence-based medicine on an individual as well as group level.y Use of novel, patient-centered and personalized outcome measures addressing (clinically) relevant items for the patient and caregivers.Annelieke Muller sHL.indd 79 14-11-2023 09:07