2 results
Approved WMORecruiting
Primary ObjectiveThe primary objective of the study is to determine the effect of ND0612 on daily *ON* time without troublesome dyskinesia (defined as the sum of "ON" time without dyskinesia and *ON* time with non-troublesome dyskinesia)…
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…