Gene therapy hope for pompe disease patients in early safety trial
NCT ID NCT03533673
First seen Jan 07, 2026 · Last updated May 15, 2026 · Updated 20 times
Summary
This early-stage study tested the safety of a new gene therapy called ACTUS-101 in 7 adults with late-onset Pompe disease. The treatment aims to help the body produce an enzyme it lacks, potentially reducing the need for regular infusions. The study focused on side effects and lab changes, not on curing the disease.
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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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Duke University
Durham, North Carolina, 27705, United States
Conditions
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