Pompe disease drug trial halted after only 3 patients
Disease control
Terminated
This study tested an oral drug called duvoglustat (AT2220) in adults with Pompe disease, a rare genetic disorder that causes muscle weakness. The goal was to see if the drug was safe and how it affected the body. Only 3 people took part, and the study was stopped early.
Phase: PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 17, 2026 15:15 UTC