Systematic Review of N-of-1 Studies in Rare Genetic Neurodevelopmental Disorders: The Power of 1.
Annelieke R MüllerMarion M M G BrandsPeter M van de VenKit C B RoesMartina Cornelia CornelClara D M van KarnebeekFrits A WijburgJoost G DaamsErik BootAgnies M van EeghenPublished in: Neurology (2021)
N-of-1 studies are sporadically reported in rare genetic neurodevelopmental disorders. Properly executed N-of-1 studies may provide a powerful alternative to larger randomized controlled trials in rare disorders and a much needed bridge between practice and science. We provide recommendations for future N-of-1 studies in rare genetic neurodevelopmental disorders, ultimately optimizing evidence-based and personalized care.