3 results
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…
Primary ObjectiveThe primary objective of the study is to determine the effect of ND0612 on daily *ON* time without troublesome dyskinesia (defined as the sum of "ON" time without dyskinesia and *ON* time with non-troublesome dyskinesia)…
OBJECTIVESPrimary ObjectivesThe primary objective is:* To assess the safety and tolerability of 6 weeks of treatment with RO7020531 administered orally to virologically suppressed chronic hepatitis B (CHB) patients.Secondary ObjectivesThe secondary…