3 results
Approved WMOCompleted
Primary Core: to demonstrate the efficacy of BAF312 relative to placebo in delaying the time to 3-month confirmed disability progression, measured by EDSS. Main secondary objectives Core: to demonstrate the efficacy in delaying the time to 3-month…
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
To explore the efficacy of central thalamic DBS in restoring consciousness in MCS patients. Hypothesis: Central thalamic DBS improves the level of consciousness in MCS patients.