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An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment with ORL-1B. | Other | Pediatric patients with biotinidase deficiency. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ORL-1B | Drug | Oral ORL-1B |
|
| Measure | Description | Time Frame |
|---|---|---|
| Improvement in seizure frequency | Statistically significant improvement in seizure frequency after the ORL-1B treatment. | 12 months |
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Inclusion Criteria:
Exclusion Criteria:
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| ID | Term |
|---|---|
| D028921 | Biotinidase Deficiency |
| ID | Term |
|---|---|
| D009100 | Multiple Carboxylase Deficiency |
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
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| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |