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 WMORecruiting
The primary objective of this exploratory proof of concept clinical investigation is to confirm bulb catheter*s performance in adults already using Navina Smart.The secondary objectives and endpoints are to evaluate bowel emptying performance of…