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A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials.

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第一作者： Barbara K,Burton

第一单位： Ann & Robert H. Lurie Children's Hospital, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.

作者： Barbara K,Burton ^[1] ; Anne,Skalicky ^[2] ; Christoph,Baerwald ^[3] ; Deborah A,Bilder ^[4] ; Cary O,Harding ^[5] ; Aaron B,Ilan ^[6] ; Elaina,Jurecki ^[6] ; Nicola,Longo ^[7] ; David T,Madden ^[6] ; H Serap,Sivri ^[8] ; Gisela,Wilcox ^[9] ; Janet,Thomas ^[10] ; Kathleen,Delaney ^[6]

作者单位： Ann & Robert H. Lurie Children's Hospital, Northwestern University Feinberg School of Medicine, Chicago, IL, USA. ^[1] Patient-Centered Research, Evidera, Seattle, WA, USA. ^[2] Rheumatology Unit, Department of Internal Medicine, Neurology and Dermatology, University Medical Center Leipzig, Leipzig, Germany. ^[3] Department of Psychiatry, Division of Child & Adolescent Psychiatry, Huntsman Mental Health Institute, University of Utah, Salt Lake City, UT, USA. ^[4] Department of Medical and Molecular Genetics, Oregon Health & Science University, Portland, OR, USA. ^[5] BioMarin Pharmaceutical Inc., Novato, CA, USA. ^[6] Division of Medical Genetics, Department of Pediatrics, University of Utah, Salt Lake City, UT, USA. ^[7] Hacettepe University Faculty of Medicine, Division of Pediatric Metabolism, Ankara, Turkey. ^[8] School of Medical Sciences, University of Manchester & The Mark Holland Metabolic Unit, Salford Royal NHS Foundation Trust, Salford, UK. ^[9] Department of Pediatrics, Section of Clinical Genetics and Metabolism, University of Colorado School of Medicine, Aurora, CO, USA. ^[10]

关键词 ADHD RS-IV, Attention Deficit Hyperactivity Disorders Rating Scale-IV ADHD, Attention Deficit Hyperactivity Disorder Adults CAARS-O:Long, Conners' Adult ADHD Rating Scales-Observer Report Long Version CGI, Clinician Global Impression COAs, Clinical Outcome Assessments COIs, Concepts Of Interests ClinROs, Clinician-Reported Outcomes Clinical outcome assessments Clinician-reported outcomes FDA, Food and Drug Administration HRQoL, Health-Related Quality of Life IRB, Institutional Review Board Neuro-QoL, Quality of Life in Neurological Disorders OGI, Observer Global Impression ObsROs, Observer-Reported Outcomes Observer-reported outcomes PAH, Phenylalanine Hydroxylase PGI, Patient Global Impression PKU PKU, Phenylketonuria PKU-QOL, PKU Quality of Life PROMIS, Patient Reported Outcome Measurement Information System PROs, Patient-Reported Outcomes Patient-reported outcomes Phe, Phenylalanine Phenylketonuria QoL, Quality of Life

DOI 10.1016/j.ymgmr.2021.100810

PMID 34815941

发布时间 2023-08-04

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Molecular genetics and metabolism reports

2021年29卷

100810页

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Molecular genetics and metabolism reports

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A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials.

Molecular genetics and metabolism reports

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A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials.

Molecular genetics and metabolism reports

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Molecular genetics and metabolism reports

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