Pompe disease drug trial halted after just 3 patients
Disease control
Terminated
This study tested an oral drug called duvoglustat in adults with Pompe disease, a rare genetic disorder that causes muscle weakness. Only 3 people took part, and the study was stopped early. Researchers looked at safety and whether the drug could improve walking distance and othe…
Phase: PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 26, 2026 16:43 UTC