3 results
Primary:* To investigate the safety and tolerability of the co-administration of GSK598809 at175 mg and ethanol in healthy volunteers.* To examine the potential pharmacokinetic interactions between GSK598809administered at 175mg and ethanol in…
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)…