3 results
The objective of the Simbici project is to evaluate the effect of bilateral cochlear implantation compared to unilateral cochlear implantation in children, in the domains of primary auditive capacities, language development and social-emotional…
Primary objective of the study is•To investigate the change of Urinary Albumin-to-Creatinine ratio (UCAR) after 90 days treatment Secondary objectives of the study are•To assess safety and tolerability of these doses by assessing the effects on…
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…