5 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 primary objective of this study is to evaluate the proportion of patients achieving FXI inhibition * 80% at trough (Day 91) after monthly dosing at 3 dose levels of MAA868.
The objective of this extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704.
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…
Primary objective:• Systemic immunogenicity of the DCP-001 vaccination in high grade serous ovarian (HGSOC) cancer patients.