2 results
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 WMOCompleted
The overall objective of the CAB LA + RPV LA clinical development programme is to develop a highly effective, well-tolerated, two-drug, long-acting injectable regimen which has the potential to offer improved treatment convenience, compliance and…