First-in-Human gene therapy tested for rare muscle disease
NCT ID NCT03533673
Summary
This is an early-stage safety study testing a new gene therapy called ACTUS-101 for adults with late-onset Pompe disease. The therapy aims to deliver a working copy of a missing gene to the liver to help the body produce a needed enzyme. The study will monitor seven participants for safety and early signs of whether the treatment is working in their muscles and lungs.
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Contacts and locations
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Locations
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Duke University
Durham, North Carolina, 27705, United States
Conditions
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