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The primary objective of this study is to evaluate potential biomarkers of GSD III.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| No Intervention | Other | No Intervention |
| Measure | Description | Time Frame |
|---|---|---|
| Urine hexose tetrasaccharide (Hex4): mean and variance | Up to Day 35 |
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Inclusion Criteria:
Exclusion Criteria:
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Subjects with a confirmed diagnosis of GSD III
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California | Irvine | California | 92697 | United States | ||
| Children's Hospital Orange County |
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| Label | URL |
|---|---|
| Ultragenyx Patient Advocacy/GSD III Disease Information | View source |
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| Orange |
| California |
| 92868 |
| United States |
| Colorado Children's Hospital | Aurora | Colorado | 80045 | United States |
| University of Texas Medical School | Houston | Texas | 77030 | United States |
| ID | Term |
|---|---|
| D006010 | Glycogen Storage Disease Type III |
| ID | Term |
|---|---|
| D006008 | Glycogen Storage Disease |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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