3 results
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…
Cohort ATo compare the CRR for the combination of pembrolizumab + BCG versus BCG alone in participants with CIS.Cohort B- To compare the EFS between participants who receive pembrolizumab + BCG (reduced maintenance) versus BCG alone- To compare the…