POMPE DISEASE

Clinical trials for POMPE DISEASE explained in plain language.

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Trials to join now! 8 Not yet finished but already full! 3 Completed 4 Terminated 1

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…

Matched conditions: POMPE DISEASE

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

Last updated Jun 26, 2026 16:43 UTC

POMPE DISEASE

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Pompe disease drug trial halted after just 3 patients