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| Name | Class |
|---|---|
| BioMarin Pharmaceutical | INDUSTRY |
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To validate the outcome measures and the tolerability of Kuvan treatment in the improvement of behavioral symptoms in 10 selected adults with Phenylketonuria (PKU) with or without mental retardation.
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| Measure | Description | Time Frame |
|---|---|---|
| Pattern of change from baseline to 6 and 12 months in Vineland scale standard scores, evaluated with a repeated measure ANOVA approach. | One year |
| Measure | Description | Time Frame |
|---|---|---|
| Correlation and regression methods (non-linear if appropriate) to examine the relationship between changes in behavior and in amino acid profiles. | one year |
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Inclusion Criteria:
Exclusion Criteria:
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Individuals with PKU followed at our clinic
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| Name | Affiliation | Role |
|---|---|---|
| Shoji Yano, MD, Ph.D. | University of Southern California | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| LAC+USC Medical Center | Los Angeles | California | 90033 | United States |
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| ID | Term |
|---|---|
| D010661 | Phenylketonurias |
| ID | Term |
|---|---|
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| D009422 | Nervous System Diseases |
| D000592 | Amino Acid 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 |