Gene therapy hope for rare childhood disease AGU
NCT ID NCT07530796
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a single dose of a gene therapy called DANAGALEX in 9 adults and children with aspartylglucosaminuria (AGU), a rare genetic disorder. The goal is to see if the treatment is safe and can reduce harmful substances in the body. Researchers will monitor side effects and measure changes in enzyme activity and blood markers.
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Conditions
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