2 results
Approved WMORecruiting
Currently, CMR examinations often contain non-evaluable sequences not suited for diagnosis due patient motion resulting from failure to maintain breath-hold. By reducing scan time and increasing patient comfort, diagnostic value will increase at…
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…