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 purpose of this study is to assess the effectiveness of extended treatment with CC compared to treatment with gonadotropins and/or the use of intra-uterine insemination (IUI), in patients who had six ovulatory cycles after CC, but did not…
The aim of the study is to determine whether cycle day (CD) 5 start of stimulation will lead to better quality of embryos, based on morphology, than CD 2 start, in IVF with GnRH antagonist co-treatment started on a fixed day.
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:To assess the safety and tolerability of single doses of CSL889 administered by IV infusion in subjects with stable SCD and in subjects with SCD in VOCSecondary Objectives:1. To characterize the PK profile of CSL889 after single IV…