Drug Information
| Drug General Information | Top | |||
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| Drug ID |
D04BQV
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| Former ID |
DCL000138
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| Drug Name |
Isofagomine tartrate
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| Synonyms |
Plicera; Afegostat tartrate; AT-2101; HGT-3410; Plicera (TN); NN-42-1007
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| Drug Type |
Small molecular drug
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| Indication | Metabolic disorder [ICD-11: 5C50-5D2Z; ICD-10: E70-E89; ICD-9: 270-279] | Phase 2 | [1], [2] | |
| Company |
Amicus Therape.
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| Structure |
 |
Download2D MOL
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| Formula |
C10H19NO9
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| Canonical SMILES |
C1C(C(C(CN1)O)O)CO.C(C(C(=O)O)O)(C(=O)O)O
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| InChI |
1S/C6H13NO3.C4H6O6/c8-3-4-1-7-2-5(9)6(4)10;5-1(3(7)8)2(6)4(9)10/h4-10H,1-3H2;1-2,5-6H,(H,7,8)(H,9,10)/t4-,5-,6-;1-,2-/m11/s1
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| InChIKey |
ULBPPCHRAVUQMC-MUMXBIPUSA-N
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| CAS Number |
CAS 919364-56-0
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| PubChem Compound ID | ||||
| PubChem Substance ID |
828686, 7888335, 10300084, 15120115, 15321577, 17406556, 22394426, 26709433, 26709438, 26711916, 36889706, 46392784, 46505453, 49655208, 50423385, 57646619, 85267413, 99376249, 103485311, 104067416, 104638232, 124490316, 125264413, 126681615, 129058498, 132631499, 134220932, 134220936, 134346715, 135260934, 137406336, 160655281, 160967546, 162065032, 164759498, 164761286, 174861454, 174861458, 174861462, 176226349, 177899992, 178103982, 179226437, 198971374, 201499585, 210024003, 221678832, 223554868, 223661383, 224146393
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Glucosylceramidase (GBA) | Target Info | Inhibitor | [3], [4] |
| KEGG Pathway | Other glycan degradation | |||
| Sphingolipid metabolism | ||||
| Metabolic pathways | ||||
| Lysosome | ||||
| Pathwhiz Pathway | Sphingolipid Metabolism | |||
| Reactome | Glycosphingolipid metabolism | |||
| WikiPathways | Sphingolipid metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 7410). | |||
| REF 2 | Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800023168) | |||
| REF 3 | Identification of pharmacological chaperones for Gaucher disease and characterization of their effects on beta-glucocerebrosidase by hydrogen/deuterium exchange mass spectrometry. Chembiochem. 2008 Nov 3;9(16):2650-62. | |||
| REF 4 | Isofagomine induced stabilization of glucocerebrosidase. Chembiochem. 2008 Nov 3;9(16):2643-9. | |||
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