Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D03ZKM
|
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| Former ID |
DIB009902
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| Drug Name |
UX-001
|
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| Drug Type |
Small molecular drug
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| Indication | Hereditary inclusion body myositis [ICD-11: 4A41.2; ICD-10: G72.4] | Phase 3 | [1] | |
| Myopathy [ICD-11: 8C7Y] | Phase 2 | [2] | ||
| Company |
Ultragenyx Pharmaceutical
|
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| Structure |
 |
Download2D MOL |
||
| Formula |
C11H19NO9
|
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| Canonical SMILES |
CC(=O)NC1C(CC(OC1C(C(CO)O)O)(C(=O)O)O)O
|
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| InChI |
1S/C11H19NO9/c1-4(14)12-7-5(15)2-11(20,10(18)19)21-9(7)8(17)6(16)3-13/h5-9,13,15-17,20H,2-3H2,1H3,(H,12,14)(H,18,19)/t5-,6-,7+,8-,9+,11+/m0/s1
|
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| InChIKey |
SQVRNKJHWKZAKO-USOVHJGOSA-N
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| PubChem Compound ID | ||||
| PubChem Substance ID |
585493, 605414, 820140, 821549, 837160, 838063, 3139621, 7890472, 7890501, 10299625, 14776496, 24888394, 24890457, 24890650, 24891482, 29196299, 36887887, 49693719, 56365699, 56435636, 57404656, 57653055, 78603070, 87350018, 87692626, 87692628, 87692631, 92093449, 92297758, 92466447, 92708681, 93576699, 99303071, 103422425, 103958633, 104633474, 117682538, 118046014, 118499594, 119524936, 123080571, 126523098, 126700501, 126722943, 126722944, 126722945, 129275585, 135207786, 135626376, 135653718
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Bacterial Deoxy-manno-octulosonate cytidylyltransferase (Bact kdsB) | Target Info | Inhibitor | [3] |
| Influenza Neuraminidase (Influ NA) | Target Info | Inhibitor | [3] | |
| KEGG Pathway | Other glycan degradation | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA) | |||
| REF 2 | ClinicalTrials.gov (NCT01830972) An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid (ER Tablets + IR Capsules) in Patients With GNE Myopathy. U.S. National Institutes of Health. | |||
| REF 3 | How many drug targets are there Nat Rev Drug Discov. 2006 Dec;5(12):993-6. | |||
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