3 results
The aim of this study is to collect data in a structured way in order to explore the tolerability, safety and efficacy of paliperiode ER with treatment schedules which will be used in future clinical practice.
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 Objective: To evaluate the effect of kinesiotaping on number of patients with clinically meaningful pain reduction after 1 week in patients with acute traumatic injury in shoulder or chest wall, compared to sham tape and no tape (2…