1 | 291844 | - | - | - | - | [2] 246 246, 299 |
2 | ATALUREN | [1] Ataluren Ataluren | [1] D09323
D09323
| - | - | [6] 19 19, 113, 140, 246, 299, 329 |
3 | Ataluren 1000mg | [1] Ataluren Ataluren | [1] D09323
D09323
| - | - | [1] 246 246 |
4 | Ataluren 125mg | [1] Ataluren Ataluren | [1] D09323
D09323
| - | - | [1] 246 246 |
5 | Ataluren 250mg | [1] Ataluren Ataluren | [1] D09323
D09323
| - | - | [1] 246 246 |
6 | BBP-671 | - | - | - | - | [2] 245 245, 246 |
7 | Bucladesine | [1] Bucladesine Bucladesine | [1] D07546
D07546
| [16] PDE10A PDE10A, PDE11A, PDE1A, PDE1B, PDE1C, PDE2A, PDE3A, PDE3B, PDE4A, PDE4B, PDE4C, PDE4D, PDE7A, PDE7B, PDE8A, PDE8B 💬 | [18] Aldosterone synthesis and secretion Aldosterone synthesis and secretion, Apelin signaling pathway, Calcium signaling pathway, Cortisol synthesis and secretion, Cushing syndrome, Glucagon signaling pathway, Insulin signaling pathway, Metabolic pathways, Morphine addiction, Olfactory transduction, Parathyroid hormone synthesis, secretion and action, Progesterone-mediated oocyte maturation, Purine metabolism, Regulation of lipolysis in adipocytes, Renin secretion, Taste transduction, cAMP signaling pathway, cGMP-PKG signaling pathway 💬 | [1] 246 246 |
8 | Carbaglu | [1] Carglumic acid Carglumic acid | [1] D07130
D07130
| [1] CPS1 CPS1 💬 | [6] Alanine, aspartate and glutamate metabolism Alanine, aspartate and glutamate metabolism, Arginine biosynthesis, Biosynthesis of amino acids, Carbon metabolism, Metabolic pathways, Nitrogen metabolism 💬 | [2] 245 245, 246 |
9 | Carglumic Acid | [1] Carglumic acid Carglumic acid | [1] D07130
D07130
| [1] CPS1 CPS1 💬 | [6] Alanine, aspartate and glutamate metabolism Alanine, aspartate and glutamate metabolism, Arginine biosynthesis, Biosynthesis of amino acids, Carbon metabolism, Metabolic pathways, Nitrogen metabolism 💬 | [3] 245 245, 246, 251 |
10 | Carglumic Acid (Carbaglu®) | [1] Carglumic acid Carglumic acid | [1] D07130
D07130
| [1] CPS1 CPS1 💬 | [6] Alanine, aspartate and glutamate metabolism Alanine, aspartate and glutamate metabolism, Arginine biosynthesis, Biosynthesis of amino acids, Carbon metabolism, Metabolic pathways, Nitrogen metabolism 💬 | [2] 245 245, 246 |
11 | Compound 1a | - | - | - | - | [2] 246 246, 299 |
12 | HLB-001 | - | - | - | - | [1] 246 246 |
13 | HST5040 | - | - | - | - | [2] 245 245, 246 |
14 | MRNA-3704 | - | - | - | - | [1] 246 246 |
15 | MRNA-3705 | - | - | - | - | [1] 246 246 |
16 | N-Carbamylglutamate | [1] Carglumic acid Carglumic acid | [1] D07130
D07130
| [1] CPS1 CPS1 💬 | [6] Alanine, aspartate and glutamate metabolism Alanine, aspartate and glutamate metabolism, Arginine biosynthesis, Biosynthesis of amino acids, Carbon metabolism, Metabolic pathways, Nitrogen metabolism 💬 | [2] 245 245, 246 |
17 | Other: standard of care | - | - | - | - | [7] 13 13, 49, 64, 85, 96, 245, 246 |
18 | PTC-0161480 | - | - | - | - | [2] 246 246, 299 |
19 | PTC-124 | - | - | - | - | [2] 246 246, 299 |
20 | PTC-C124 | - | - | - | - | [2] 246 246, 299 |
21 | PTC124 | - | - | - | - | [4] 19 19, 113, 246, 299 |
22 | RPS 2505 | - | - | - | - | [2] 246 246, 299 |
23 | Standard of Care Treatment | - | - | - | - | [3] 53 53, 245, 246 |