Navigating the outcome maze: a scoping review of outcomes and instruments in clinical trials in genetic neurodevelopmental disorders and intellectual disability.

Autor: Müller AR; Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands.; Advisium, 's Heeren Loo, Amersfoort, Utrecht, The Netherlands.; Amsterdam Public Health Research Institute, Amsterdam, The Netherlands.; Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, The Netherlands., van Silfhout NY; Amsterdam Public Health Research Institute, Amsterdam, The Netherlands.; Amsterdam Reproduction & Development, Child Development, Amsterdam, The Netherlands.; Emma Children's Hospital, Child and Adolescent Psychiatry & Psychosocial Care, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands., den Hollander B; Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands.; Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, The Netherlands.; United for Metabolic Diseases, Amsterdam, The Netherlands., Kampman DHC; Faculty of Science, Operational Management, ICT Department, Utrecht University, Utrecht, The Netherlands., Bakkum L; Amsterdam Public Health Research Institute, Amsterdam, The Netherlands.; Department of Clinical Child and Family Studies, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands., Brands MMMG; Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands.; Emma Center for Personalized Medicine, Amsterdam UMC, Amsterdam, The Netherlands.; Amsterdam Reproduction & Development, Child Development, Amsterdam, The Netherlands.; United for Metabolic Diseases, Amsterdam, The Netherlands., Haverman L; Amsterdam Public Health Research Institute, Amsterdam, The Netherlands.; Amsterdam Reproduction & Development, Child Development, Amsterdam, The Netherlands.; Emma Children's Hospital, Child and Adolescent Psychiatry & Psychosocial Care, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands., Terwee CB; Amsterdam Public Health Research Institute, Amsterdam, The Netherlands.; Epidemiology and Data Science, Amsterdam UMC Location Vrije Universiteit, Amsterdam, The Netherlands., Schuengel C; Amsterdam Public Health Research Institute, Amsterdam, The Netherlands.; Department of Clinical Child and Family Studies, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands., Daams J; Medical Library, Research Support, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands., Hessl D; MIND Institute and Department of Psychiatry and Behavioral Sciences, University of California Davis, Sacramento, CA, USA., Wijburg FA; Department of Pediatrics, Emma Children's Hospital, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, Amsterdam, The Netherlands., Boot E; Advisium, 's Heeren Loo, Amersfoort, Utrecht, The Netherlands.; The Dalglish Family 22q Clinic, Toronto, ON, Canada.; Department of Psychiatry & Neuropsychology, Maastricht University Medical Center, Maastricht, The Netherlands., van Eeghen AM; Emma Center for Personalized Medicine, Amsterdam UMC, Meibergdreef 9, Amsterdam, 1105 AZ, The Netherlands.; Advisium, 's Heeren Loo, Berkenweg 11, 3818 LA, Amersfoort, The Netherlands.; Amsterdam Public Health Research Institute, Meibergdreef 9, 1105 AZ, Amsterdam, The Netherlands.; Amsterdam Reproduction & Development, Child Development, Meibergdreef 9, 1105 AZ, Amsterdam, The Netherlands.

Publikováno v: Therapeutic advances in rare disease [Ther Adv Rare Dis] 2024 Apr 25; Vol. 5, pp. 26330040241245721. Date of Electronic Publication: 2024 Apr 25 (Print Publication: 2024).