Pivotal phase gene transfer clinical trial for Mucopolysaccharidosis Type IIIA delivering LYS-SAF302

Pivotal phase gene transfer clinical trial for Mucopolysaccharidosis Type IIIA delivering LYS-SAF302 What is the purpose of this study? Lysogene is preparing a pivotal phase 2-3 clinical trial to assess the efficacy of direct to CNS delivery of LYS-SAF302 in improving or stabilizing the neurodevelopmental status of MPS IIIA patients. The trial will start in the [...]