6 results
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…
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 gather more information about the long-term effect of enzyme therapy in patients with Pompe disease with a different severity of disease. The goal is also to set guideline for start and stop of treatment and to…
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…
To assess whether the use of the personalized risk assessment tool (PERSARC) is (cost)effective in reducing decisional conflict and increasing informed choices in high-grade extremity STS compared to usual care (co-primary outcomes). In addition, we…
The central aim of the POEM Study: to investigate whether the early start of metformin on top of standard care may improve the treatment of GDM for mother and child. Metformin is used as a tool to reduce insulin resistance, the underlying cause of…