Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
Status:
Terminated
Trial end date:
2009-12-14
Target enrollment:
Participant gender:
Summary
The main purpose of this study was to determine the safety and tolerability of 3 different
doses of duvoglustat (AT2220) in participants affected by Pompe disease. The study also
evaluated the effects of duvoglustat on functional parameters in Pompe disease.