Pompe disease drug trial halted after only 3 patients
NCT ID NCT00688597
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 30 times
Summary
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.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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Study site
Decatur, Georgia, 30033, United States
Conditions
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