Qualitative research to explore and support the assessment of the patient experience and impact of functioning of movement disorders in glucose transporter type 1 deficiency syndrome, and literature review in absence seizures
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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