3 results
Approved WMOPending
Primary Objective: To investigate the effect on the distention of the ONSD while using lidocaine 1,5 mg/kg IV during endotracheal intubation versus placebo. Secondary Objectives: The secondary objective of this study is to investigate the effect…
Approved WMOWill not start
Primary* To estimate the effect of NME combination therapies on inducing a functional cure over the control arm.Secondary * To characterize the efficacy profile of NME combination therapies.* To characterize the PD profile of NME combination…
Approved WMORecruiting
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.