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Aspartylglucosaminuria

MONDO:0008830

Aspartylglycosaminuria (AGU) is an autosomal recessive lysosomal storage disease belonging to the oligosaccharidosis group (also called glycoproteinosis).

Also known as: Aspartylglycosaminuria, aspartylglucosaminidase deficiency, aspartylglucosaminuria, aspartylglycosaminuria, glycosylasparaginase deficiency, AGU, Aga deficiency, Aspartylglucosamidase (AGA) deficiency

4 clinical trials for this condition and its sub-types.

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Broader categories

Disease (680) Metabolic disease (233) Musculoskeletal system disorder (207) Hereditary disease (176) Bone disorder (51) Inborn errors of metabolism (45) Lysosomal storage disease (35) Human disease (14) Developmental defect during embryogenesis (8) Skeletal system disorder (4)
Trials to join now! 1 Not yet recruiting 1 Not yet finished but already full! 2
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  • Gene therapy hope for rare childhood disease AGU

    Disease control Not yet recruiting

    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 s…

    Phase: PHASE1, PHASE2 • Sponsor: Rare Trait Hope • Aim: Disease control

    Last updated Jun 27, 2026 12:07 UTC

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