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Table of contentsChapter 1 General introduction 9PART I N-of-1 studies in rare genetic neurodevelopmental disorders27Chapter 2 Systematic review of N-of-1 studies in rare genetic neurodevelopmental disorders: the power of 129Chapter 3 Methylphenidate for attention-deficit/hyperactivity disorder in patients with Smith-Magenis syndrome: protocol for a series of N-of-1 trials55Chapter 4 Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with Tuberous Sclerosis Complex, mucopolysaccharidosis type III and Fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials77PART II Outcome measures for rare genetic neurodevelopmental disorders and intellectual disability107Chapter 5 Navigating the outcome maze: A scoping review of outcomes and instruments in clinical trials in genetic neurodevelopmental disorders and intellectual disability109Chapter 6 Understanding the impact of tuberous sclerosis complex: development and validation of the TSCPROM137Annelieke Muller sHL.indd 6 14-11-2023 09:07