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http://dx.doi.org/10.1097/BRS.0000000000004725 | DOI Listing |
Trials
December 2024
School of Medicine Depts of Pediatrics, Neurology and Pharmacology, Children's Hospital Colorado/University of Colorado, 12800 E 19th, MS8102, Aurora, CO, 80045, USA.
Introduction: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.
Methods: Clinical outcome assessments for the top 10 priority domains identified in the Voice of the Patient Report for Rett syndrome were compiled and available psychometric data were extracted.
Lancet
December 2024
Department of Child Dental Health, Obafemi Awolowo University, Ile-Ife, Nigeria.
J Clin Anesth
December 2024
Department of Anesthesiology, Perioperative Care and Pain Medicine, NYU Langone Health, 550 First Avenue, New York, NY 10016, USA. Electronic address:
Trends Cogn Sci
December 2024
Institute of Philosophy, School of Advanced Study, University of London, London, UK. Electronic address:
Am J Emerg Med
December 2024
Department of Emergency Medicine, Hamad Medical Corporation, Hamad General Hospital, Doha, Qatar; Department of Emergency Medicine, Blizard Institute, Queen Mary University, London, UK. Electronic address:
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