3 results
Approved WMOWill not start
Peroperative assessment of the heart's microcirculation during coronary arterial bypass surgery (CABG) with incident dark filed imaging
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…
Approved WMORecruiting
The primary objectives of the study are to:Determine the OBD(s) and RP2D's) of BMF-219 monotherapy administered daily based on evaluation of all available PK/ PD, target engagement, safety, and tolerability data. Note that the OBD and RP2D may…