2 results
Approved WMOCompleted
To assess the effect of AMG785 treatment for 12 months followed by alendronate treatment compared with alendronate on the incidence of clinical fracture and new vertebral fracture.
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…