3 results
Approved WMOCompleted
The overall objective of this study is to evaluate the efficacy and safety of neoGAA in treatment-naïve patients with LOPD as compared to alglucosidase alfa, when this is administered biweekly for a period of 49 weeks. Also, there is an open-label…
Approved WMORecruiting
The objective of the study is to asses the efficacy and safety of intravenous ATB200 co-administration with oral AT2221 by evaluating the changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset…
Approved WMOPending
Primary:To assess the safety and tolerability of DS-1211b compared with placebo in subjects with PXE.To assess the dose response by assessing the treatment changes in PD endpoints.