3 results
Approved WMOPending
To establish the safety and tolerablity of multiple doses of hLF1-11 given once daily for 10 days.
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…
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)…