Details of the Drug

General Information of Drug (ID: DM7RHOC)

• Drug Name: 5N,6S-(N'-butyliminomethylidene)-6-thio-1-deoxygalactonojirimycin

Indication

Disease Entry	ICD 11	Status	REF
GM1 gangliosidosis	5C56.00	Preclinical	[1]

• Drug Type: Small molecular drug
• Cross-matching ID:
  PubChem CID: 76070514
  TTD ID: D2DLN9

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Beta-galactosidase (GLB1)	TTNGJPH	BGAL_HUMAN	Inhibitor	[2]

References

• [1] Lysosomes as a therapeutic target. Nat Rev Drug Discov. 2019 Dec;18(12):923-948.
• [2] A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis. Mol Ther. 2013 Mar;21(3):526-32.