4 results
To evaluate the safety and tolerability of FFP104 in PBC subjects following repeat doses of FFP104
To evaluate the safety and efficacy of intravenously administered FFP104 in subjects with active Crohn*s disease following repeat doses of FFP104.
Primary:To estimate the treatment effect of emactuzumab on objective response rate (ORR) by 6 months from initiation of therapy in the blinded phase compared to placeboSecondary:the effect of emactuzumab on clinical outcome assessments (COAs) for:o…
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…