Argininosuccinate Synthetase Deficiency (Citrullinemia)
Also known as: Argininosuccinic acid synthetase deficiency / Citrullinemia / Argininosuccinate Synthetase Deficient / Argininosuccinate synthetase deficiency / Citrullinemia (disorder) / Citrullinaemia / Deficiency of argininosuccinate synthase (disorder)
| 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 |
|---|---|---|---|---|
| DB06592 | Pegargiminase | 1 | Completed | 1 |
| DB00551 | Acetohydroxamic acid | 1 / 2 | Not Yet Recruiting | 1 |
| DB03904 | Urea | 1 / 2 | Not Yet Recruiting | 1 |
| DB17695 | Heterologous human adult liver derived progenitor cells | 2 | Completed | 1 |