Oral pill tested to fight rare muscle disease
NCT ID NCT00688597
Summary
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 ability. The study was small and ended early, and the drug was designed to help manage the disease, not cure it.
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Contacts and locations
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Locations
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Study site
Decatur, Georgia, 30033, United States
Conditions
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