2 results
Approved WMORecruiting
The main objective is to investigate if the use of 5mm needles has a positive effect concerning hypoglycemic events and local complaints like bruising, bleeding, back flow of insulin and pain compared to 8mm needles, and to receive information about…
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…