2 results
Approved WMORecruiting
Are there differences is efficacy, side effects, tolerance en costs in patients with early RA in treatment with COBRA-light compared with COBRA according to BeSt?
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…