One-Shot gene therapy trial offers new hope for pompe disease
NCT ID NCT04174105
Summary
This early-stage study is testing a one-time gene therapy called AT845 for adults with late-onset Pompe disease. The goal is to see if delivering a working copy of the faulty gene directly into the body is safe and can help control the disease. Participants receive a single infusion and are followed for up to 10 years to monitor their health and how well the treatment works.
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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