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

New hope for pompe patients: first human trial tests promising drug combo

Disease control Completed

This study tested whether a new two-drug combination (ATB200 given by IV plus AT2221 taken by mouth) is safe and effective for adults with Pompe disease. Researchers enrolled 29 adults, including both those who had previously received standard enzyme replacement therapy and those…

Matched conditions: POMPE DISEASE

Phase: PHASE1, PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control

Last updated Mar 30, 2026 14:29 UTC

POMPE DISEASE

Get alerted when new POMPE DISEASE trials appear

New hope for pompe patients: first human trial tests promising drug combo