3 results
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…
We hypothesize that the timing of treatment in IA, also known as chronotherapy, matters and that the efficacy of tofacitinib XR depends on it.Therefore, the aims of this randomized controlled trial are: 1. To compare the clinical effectiveness of…