8 results
Primary: Safety and tolerability of 2 dosages of ambrisentan. Secundary: Efficacy and PK.
Primary: Longterm safety and tolerability of 2 dosages of ambrisentan. Secundary: Supportive efficacy data.
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.
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 compare the two treatment strategies; first-line combination therapy (ambrisentan and tadalafil) versus first-line monotherapy (ambrisentan or tadalafil) in subjects with PAH. This will be assessed by time…
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: to assess the efficacy of ambrisentan 5mg after treatment period of 16 weeks, in subjects with inoperable CTEPH. Secondary: safety and tolerability.
Primary: to assess the longterm safety and tolerability of ambrisentan 5mg in subjects with inoperable CTEPH. Secondary: to collect supportive efficacy data.