| No. |
Drug (Description in trials) |
DrugBank |
KEGG DRUG * |
KEGG GENES * [Number of genes] |
KEGG PATHWAY [Number of pathways] |
Disease ID * [Number of diseases] |
| 1 |
291844 |
- |
- |
- |
- |
[2] 246, 299 |
| 2 |
Ataluren |
Ataluren |
D09323 |
- |
- |
[6] 19, 113, 140, 246, 299, 329 |
| 3 |
Ataluren 1000mg |
Ataluren |
D09323 |
- |
- |
[1] 246 |
| 4 |
Ataluren 125mg |
Ataluren |
D09323 |
- |
- |
[1] 246 |
| 5 |
Ataluren 250mg |
Ataluren |
D09323 |
- |
- |
[1] 246 |
| 6 |
Carbaglu |
Carglumic acid |
D07130 |
[1] CPS1 |
[6] Alanine, aspartate and glutamate metabolism, Arginine biosynthesis, Biosynthesis of amino acids, Carbon metabolism, Metabolic pathways, Nitrogen metabolism |
[2] 245, 246 |
| 7 |
Carglumic acid |
Carglumic acid |
D07130 |
[1] CPS1 |
[6] Alanine, aspartate and glutamate metabolism, Arginine biosynthesis, Biosynthesis of amino acids, Carbon metabolism, Metabolic pathways, Nitrogen metabolism |
[2] 245, 246 |
| 8 |
Compound 1a |
- |
- |
- |
- |
[2] 246, 299 |
| 9 |
Mrna-3704 |
- |
- |
- |
- |
[1] 246 |
| 10 |
N-carbamylglutamate |
Carglumic acid |
D07130 |
[1] CPS1 |
[6] Alanine, aspartate and glutamate metabolism, Arginine biosynthesis, Biosynthesis of amino acids, Carbon metabolism, Metabolic pathways, Nitrogen metabolism |
[2] 245, 246 |
| 11 |
Ptc-0161480 |
- |
- |
- |
- |
[2] 246, 299 |
| 12 |
Ptc-124 |
- |
- |
- |
- |
[2] 246, 299 |
| 13 |
Ptc-c124 |
- |
- |
- |
- |
[2] 246, 299 |
| 14 |
Ptc124 |
- |
- |
- |
- |
[4] 19, 113, 246, 299 |
| 15 |
Rps 2505 |
- |
- |
- |
- |
[2] 246, 299 |
| 16 |
Standard of care treatment |
- |
- |
- |
- |
[2] 245, 246 |