POMPE DISEASE

Clinical trials for POMPE DISEASE explained in plain language.

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Trials to join now! 7 Not yet finished but already full! 2 Completed 2 Terminated 1

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.

Matched conditions: POMPE DISEASE

Phase: PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control

Last updated May 17, 2026 11:37 UTC

POMPE DISEASE

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Pompe disease drug trial halted after only 3 patients