Carbamyl Phosphate Synthetase Deficiency
Also known as: Carbamylphosphate synthetase deficiency / Carbamyl phosphate synthetase / Carbamoyl-phosphate synthetase 1 deficiency (disorder) / Carbamoyl phosphate synthetase deficiency / Carbamoyl-Phosphate Synthase I Deficiency Disease
| Drug | Drug Name | Drug Description |
|---|---|---|
| DB06819 | Phenylbutyric acid | An agent indicated for the adjunctive therapy for the management of chronic urea cycle disorders due to deficiencies in specific enzymes, including the neonatal-onset deficiency and late-onset disease with a history of hyperammonemic encephalopathy. |
| Drug | Drug Name | Target | Type |
|---|---|---|---|
| DB06819 | Phenylbutyric acid | Aromatic-amino-acid aminotransferase | target |
| DB06819 | Phenylbutyric acid | Histone deacetylase | target |
| Drug | Drug Name | Phase | Status | Count |
|---|---|---|---|---|
| DB06775 | Carglumic acid | 2 | Withdrawn | 1 |