Gene therapy hope for rare AGU disease enters human trials
NCT ID NCT07530796
First seen Apr 24, 2026 · Last updated May 12, 2026 · Updated 5 times
Summary
This early-stage trial tests a single-dose gene therapy called DANAGALEX in 9 adults and children with AGU, a rare genetic disorder. The goal is to see if it is safe and can reduce harmful substances in the body. Participants must have a confirmed AGU diagnosis and be enrolled in a natural history study.
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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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