New gene therapy shows promise for rare muscle disease
NCT ID NCT03533673
First seen Jan 07, 2026 · Last updated May 25, 2026 · Updated 21 times
Summary
This early-stage study tested a gene therapy called ACTUS-101 in 7 adults with late-onset Pompe disease, a condition that causes muscle weakness. The main goal was to check safety and see if the treatment could increase the missing enzyme. Participants received a single intravenous dose, and researchers monitored side effects and enzyme levels over time.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Duke University
Durham, North Carolina, 27705, United States
Conditions
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