Page 25 - Demo
P. 25
General introduction 23147. Mitchell GK, Hardy JR, Nikles CJ, et al. The effect of methylphenidate on fatigue in advanced cancer: An aggregated N-of-1 trial. J. Pain Symptom Manage. 2015.48. Bradbury J, Avila C, Grace S. Practice-Based Research in Complementary Medicine: Could N-of-1 Trials Become the New Gold Standard? Healthcare 2020.49. Goetz LH, Schork NJ. Personalized medicine: motivation, challenges, and progress. Fertil. Steril. 2018;50. Schork NJ. Personalized medicine: Time for one-person trials. Nature 2015.51. Beckers LWME, Stal RA, Smeets RJEM, et al. Single-case Design Studies in Children with Cerebral Palsy: A Scoping Review. Dev. Neurorehabil. 2020.52. Evans JJ, Gast DL, Perdices M, Manolov R. Single case experimental designs: Introduction to a special issue of Neuropsychological Rehabilitation. Neuropsychol. Rehabil. 2014.53. Smith JD. Single-case experimental designs: A systematic review of published research and current standards. Psychol. Methods 2012.54. Weinreich SS, Vrinten C, Kuijpers MR, et al. Aggregated N-of-1 trials for unlicensed medicines for small populations: an assessment of a trial with ephedrine for myasthenia gravis. Orphanet J. Rare Dis. 2017.55. Hessl D, Sansone SM, Berry-Kravis E, et al. The NIH Toolbox Cognitive Battery for intellectual disabilities: Three preliminary studies and future directions. J. Neurodev. Disord. 2016;8(1).56. Berry-Kravis EM, Hessl D, Rathmell B, et al. Effects of STX209 (Arbaclofen) on neurobehavioral function in children and adults with fragile X syndrome: A randomized, controlled, phase 2 trial. Sci. Transl. Med. 2012;4(152).57. Jacquemont S, Curie A, Des Portes V, et al. Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056. Sci. Transl. Med. 2011;3(64).58. Budimirovic DB, Berry-Kravis E, Erickson CA, et al. Updated report on tools to measure outcomes of clinical trials in fragile X syndrome. J. Neurodev. Disord. 2017.59. Shields RH, Kaat AJ, McKenzie FJ, et al. Validation of the NIH Toolbox Cognitive Battery in intellectual disability. Neurology 2020.60. Dumont LJ, Aubuchon JP. Evaluation of proposed FDA criteria for the evaluation of radiolabeled red cell recovery trials. Transfusion 2008;48(6).61. World Health Organization. Towards a Common Language for Functioning , Disability and Health ICF. Int. Classif. 2002.62. Frew JW, Davidson M, Murrell DF. Disease-specific health related quality of life patient reported outcome measures in Genodermatoses: A systematic review and critical evaluation. Orphanet J. Rare Dis. 2017.63. Thompson JJ, Kelly KL, Ritenbaugh C, et al. Developing a patient-centered outcome measure for complementary and alternative medicine therapies II: Refining content validity through cognitive interviews. BMC Complement. Altern. Med. 2011;11.64. Valderas JM, Kotzeva A, Espallargues M, et al. The impact of measuring patient-reported outcomes in clinical practice: A systematic review of the literature. Qual. Life Res. 2008.65. Mercieca-Bebber R, King MT, Calvert MJ, et al. The importance of patient-reported outcomes in clinical trials and strategies for future optimization. Patient Relat. Outcome Meas. 2018.66. Bijkerk CJ, De Wit NJ, Muris JWM, et al. Outcome measures in irritable bowel syndrome: Comparison of psychometric and methodological characteristics. Am. J. Gastroenterol. 2003;98(1).67. Lee EH, Kwon O, Hahm KB, et al. Irritable bowel syndrome-specific health-related quality of life instrument: Development and psychometric evaluation. Health Qual. Life Outcomes 2016;14(1).68. Van Eeghen AM, Bruining H, Wolf NI, et al. Personalized medicine for rare neurogenetic disorders: can we make it happen? Cold Spring Harb Mol Case Stud 2022;8(2):a006200.69. Srikrishna S, Robinson D, Cardozo L, Gonzalez J. Is there a discrepancy between patient and physician quality of life assessment? Neurourol. Urodyn. 2009;28(3).Annelieke Muller sHL.indd 23 14-11-2023 09:07