2 results
Approved WMOCompleted
To evaluate the safety, tolerability and pharmacokinetics of RO5428029 in healthy male subjects.
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…