Methylmalonic Acidemia
Also known as: Methylmalonic aciduria / Methylmalonic Acidemia (MMA) / Methylmalonicacidemia / Methylmalonic acidaemia / Methylmalonic acidemia (disorder)
| Drug | Drug Name | Drug Description |
|---|---|---|
| DB06775 | Carglumic acid | An analog of N-acetylglutamate (NAG) used for the treatment of acute and chronic hyperammonemia in patients with N-acetylglutamate synthase (NAGS) deficiency. |
| Drug | Drug Name | Target | Type |
|---|---|---|---|
| DB06775 | Carglumic acid | Carbamoyl-phosphate synthase [ammonia], mitochondrial | target |
| Drug | Drug Name | Phase | Status | Count |
|---|---|---|---|---|
| DB18498 | BBP-671 | 1 | Terminated | 1 |
| DB04045 | (R)-methylmalonyl-CoA | 1 / 2 | Recruiting | 2 |
| DB18434 | SEL-302 | 1 / 2 | Suspended | 1 |
| DB17668 | mRNA-3705 | 1 / 2 | Recruiting | 2 |
| DB06775 | Carglumic acid | 2 | Completed | 1 |
| DB06775 | Carglumic acid | 2 | Terminated | 1 |
| DB06775 | Carglumic acid | 2 | Withdrawn | 1 |
| DB06775 | Carglumic acid | 3 | Completed | 1 |
| DB06775 | Carglumic acid | Not Available | Enrolling by Invitation | 1 |
| DB06775 | Carglumic acid | Not Available | Recruiting | 2 |