Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D2DLN9
|
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| Drug Name |
5N,6S-(N'-butyliminomethylidene)-6-thio-1-deoxygalactonojirimycin
|
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| Drug Type |
Small molecular drug
|
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| Indication | GM1 gangliosidosis [ICD-11: 5C56.00] | Preclinical | [1] | |
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Beta-galactosidase (GLB1) | Target Info | Inhibitor | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | Lysosomes as a therapeutic target. Nat Rev Drug Discov. 2019 Dec;18(12):923-948. | |||
| REF 2 | A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis. Mol Ther. 2013 Mar;21(3):526-32. | |||
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