5 results
The primary objective of the trial is to investigate the efficacy of apomorphine subcutaneous infusion compared to placebo in PD patients with motor fluctuations not well controlled on medical treatment. The secondary objective is to investigate the…
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 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 primary objectives of the Phase Ib portion of the study are as follows:* To assess the safety and tolerability of the combination of polatuzumab vedotin with bendamustine and rituximab (BR) or bendamustine and obinutuzumab (BG) when administered…
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…