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 is to assess the feasibility of intraductal biopsy and intraductal laser ablation in patients with intraductal lesions. Secondary objectives are: treatment success of interventional ductoscopy (biopsy and laser ablation) in…