3 results
Approved WMORecruiting
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.
Approved WMOPending
To establish the safety, tolerability, and early efficacy of multiple doses of hLF1-11 given once daily for 14 days.
Approved WMOCompleted
The aim of this prospective study is 1) to evaluate the diagnostic efficacy of DBE in comparison to MRE as surveillance technique of the small-bowel in PJS patients, 2) to analyze patient burden and quality of life with DBE and MRE, and 3) to…