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Chapter 114for particular disorders including vigabatrin as a treatment for epilepsy in Tuberous Sclerosis Complex.36,37 Similarly, it implies targeted behavioral interventions, such as anticipatory care planning by behavioral experts and caregivers for individuals with Down syndrome who eventually all show neuropathological changes of Alzheimer’s disease at an early age.38Interventional research has generally performed per disorder.39 However, effectiveness of interventions has largely remained unclear, resulting in affected individuals missing out on possibly effective interventions.Trial methodology: how to do interventional research in RGNDs? Interventional research is challenging in individuals with RGNDs and ID due to the rarity of the conditions and heterogeneity of manifestations. These manifestations typically show great inter- and intraindividual variability, hampering conventional trial designs. Parallel group randomized controlled trials (RCTs), considered the gold standard for interventional studies, are often not feasible in small and heterogeneous populations. Due to strict eligibility criteria, affected individuals are often excluded. To stimulate the search for treatments of these largely ignored rare disorders, a new methodological framework needs to be developed.Single-case experimental designsSingle-case experimental designs (SCEDs) may provide a powerful solution for interventional research in RGNDs and ID, and a much-needed bridge between practice and science. SCEDs are experimental designs to test the effectiveness of an intervention for an individual participant who acts as their own control, using repeated outcome measurements and sequential or randomized introduction of the intervention.40 SCEDs may increase acceptance by clients and physicians and may facilitate recruitment. Also, SCEDs require less participants than conventional trial designs, efficiently using data from participants.41 Commonly used SCEDs include the multiple baseline design, the changing criterion design, the alternating treatments design, and the N-of-1 design.42N-of-1 trialsOf the SCEDs, the N-of-1 design provides the highest level of evidence, as it closely follows indications of causality.43 N-of-1 studies are randomized, Annelieke Muller sHL.indd 14 14-11-2023 09:07