Rare Trait Hope
Rare Trait Hope
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Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU) | Aspartylglucosamidase (AGA) deficiency, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, aspartylglucosaminuria, hereditary disease, inborn errors of metabolism, lysosomal storage disease, metabolic disease | Not yet recruiting | Phase 1 / Phase 2 | 2026-04-15 | NCT07530796 |