Development of a COA strategy for Down’s syndrome in adults and children
Clinical meaningfulness of scores on the Vineland Adaptive Behaviour Scale (VABS-II), Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) and Clinician Global Impression (CGI) in Down’s syndrome
Strategic literature review to identify COA instruments to assess the concept of independence in Down’s syndrome
Qualitative interviews and development of a COA strategy for Down’s syndrome in adults and children
Subgroup analysis by chronological age of individuals with Down’s syndrome
Exploration of IQ as a relevant endpoint in Hunter’s syndrome
Endpoint review in Hunter’s syndrome
PRO dossier on the Disease Activity Score (DAS), Scales of Independent Behaviour-Revised (SIB-R), Behaviour Rating Inventory of Executive Function (BRIEF) and Vineland Adaptive Behaviour Scales (VABS) for Hunter’s syndrome
Strategic PRO and ObsRO analysis of a Hunter’s Syndrome clinical trial database
Regulatory support for questionnaires in Hunter’s syndrome
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