6 results
Primary: To assess the efficacy of intracerebral delivery of AAVrh.10SGSH gene therapy (LYS-SAF302) in improving or stabilizing the neurodevelopmental status of MPS IIIA patients after 24 months (main cohort), compared to the expected evolution…
- To determine between-subject and within-subject variability of the study endpoints.- To determine the differences between subjects and healthy controls regarding the study endpoints.- To identify suitable endpoints for future clinical trials in…
The primary objective is to evaluate the effects of administration of neflamapimod (VX-745) for 24-weeks on immediate and delayed recall aspects of episodic memory, as assessed by the Hopkins Verbal Learning Test * Revised (HVLT-R) in subjects with…
The first objective of this study is to provide a detailed, quantitative and qualitative description of early language development (*developmental language profile*) in children with 22q11DS, comprising expressive and receptive abilities, covering…
With this study we aim to define a detailed oral motor, speech and language profile for patients with SAS.
Our primary objective is to compare children with FXS with children with non-syndromic ASD, a developmental delay (DD) and typically developing (TD) children on three domains: ASD severity, cognitive abilities and (neuro)biological characteristics.…