2 results
Approved WMOWill not start
Primary Objective:To preliminarily evaluate the efficacy of GLPG0259 compared with placebo in terms of the proportion of subjects achieving ACR20 at Week 12 (Visit [V]7).Secondary Objectives:•To evaluate the efficacy of GLPG0259 compared with…
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…