3 results
Primairy objective:• To determine the safety, tolerability and pharmacokinetics of 2B3-201, and in comparison to free methylprednisolone hemisuccinate and placebo;Secundairy objective:• To determine the pharmacodynamic effects of 2B3-201 on CNS…
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…
The aim is to evaluate the clinical performance of the system and verify the claims of reduced interference with biotin and CCDs. The measurements on NOVEOS will be mirrored in this study against the clinical diagnosis, the outcome of skin prick…