Gene therapy may reduce infusions for pompe patients
NCT ID NCT04174105
First seen Nov 01, 2025 · Last updated Apr 24, 2026 · Updated 30 times
Summary
This study tests a new gene therapy called AT845 for adults with late-onset Pompe disease. The goal is to see if a single dose can help the body produce the missing enzyme, potentially reducing or replacing the need for regular enzyme infusions. Eleven participants will receive one of several doses and be monitored for safety and muscle improvements.
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This is a summary of
the original study
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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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Contacts and locations
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Locations
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Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility
Newcastle upon Tyne, NE1 4LP, United Kingdom
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Stanford University
Palo Alto, California, 94304, United States
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University of California Irvine, Department of Neurology
Orange, California, 92868, United States
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University of Utah, Division of Medical Genetics
Salt Lake City, Utah, 84108, United States
Conditions
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