3 results
The main objective of this study is to further determine the efficacy of lapaquistat acetate 50mg compared to placebo during a period of 12 weeks. This period is followed by an optional 48 week open label extension to be able to study the long term…
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…
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…