3 results
Approved WMOCompleted
Primary ObjectiveThe primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in patients with late-onset Pompe disease.Secondary ObjectivesThe secondary…
Approved WMOPending
Evaluation of the efficacy and tolerability of adding verapamil to the antiepileptic drug regimen in patients who did not become seizure free on carbamazepine.
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…