POMPE DISEASE

Clinical trials for POMPE DISEASE explained in plain language.

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Trials to join now! 6 Not yet finished but already full! 3 Completed 1 Terminated 1

Oral pill tested to fight rare muscle disease

Disease control Terminated

This study tested the safety and effects of three different dosing schedules of an oral drug called duvoglustat in adults with Pompe disease, a rare genetic disorder that weakens muscles. The goal was to see if the drug was safe and if it could improve muscle function and walking…

Matched conditions: POMPE DISEASE

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

Last updated Mar 23, 2026 15:17 UTC

POMPE DISEASE

Get alerted when new POMPE DISEASE trials appear

Oral pill tested to fight rare muscle disease