2 results
Approved WMOCompleted
Primary objective: To examine the effects of calcitriol on the activity of dopaminergic central nervous system regions involved in the visualization and regulation of food intake using functional magnetic resonance imaging (fMRI). Secondary…
Approved WMORecruiting
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…