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Chemical Structure| 210110-90-0 Chemical Structure| 210110-90-0

Structure of Miglustat HCl
CAS No.: 210110-90-0

Chemical Structure| 210110-90-0

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Miglustat HCl is an inhibitor of glucosylceramide synthase, primarily to treat Type I Gaucher disease (GD1).

Synonyms: OGT 918 hydrochloride; N-Butyldeoxynojirimycin hydrochloride; NB-DNJ

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Product Details of Miglustat HCl

CAS No. :210110-90-0
Formula : C10H22ClNO4
M.W : 255.74
SMILES Code : O[C@@H]1[C@@H](CO)N(CCCC)C[C@H](O)[C@H]1O.[H]Cl
Synonyms :
OGT 918 hydrochloride; N-Butyldeoxynojirimycin hydrochloride; NB-DNJ
MDL No. :MFCD00269940
InChI Key :QPAFAUYWVZMWPR-ZSOUGHPYSA-N
Pubchem ID :6603107

Safety of Miglustat HCl

GHS Pictogram:
Signal Word:Warning
Hazard Statements:H361
Precautionary Statements:P501-P202-P201-P280-P308+P313-P405

Isoform Comparison

Biological Activity

In Vitro:

Cell Line
Concentration Treated Time Description References
Human astrocytes 100 μM 1 hour To study the effect of Miglustat on the inflammatory response of human astrocytes, results showed that Miglustat suppressed the expression of pro-inflammatory genes. Cell. 2019 Dec 12;179(7):1483-1498. e22
Peripheral blood mononuclear cells (PBMC) 1 mM 24 hours To evaluate the effect of Miglustat on cytokine release following SARS-CoV-2 S glycoprotein stimulation, results showed no statistically significant effects on cytokine production. J Clin Immunol. 2021 Feb;41(2):335-344
NIH3T3 cells 1 mM 24 hours To evaluate the effect of Miglustat on SARS-CoV-2 S-driven cell-to-cell fusion, results showed no statistically significant differences in syncytia formation frequency or size comparing miglustat-treated samples to untreated controls. J Clin Immunol. 2021 Feb;41(2):335-344
HEK293T cells 1 mM 24 hours To evaluate the effect of Miglustat on the N-glycosylation pattern of SARS-CoV-2 S and ACE2 glycoproteins, results showed that Miglustat successfully and specifically modified the N-glycosylation pattern of both glycoproteins. J Clin Immunol. 2021 Feb;41(2):335-344
hDA neurons 100 μM 3 days Reduced GlcCer accumulation, restored the ratio of α-syn tetramers and related multimers, and protected neurons from PFF-induced toxicity Proc Natl Acad Sci U S A. 2018 Jan 23;115(4):798-803
SH-SY5Y cells 100 μM 3 days Reduced intracellular GlcCer levels and restored the ratio of α-syn tetramers and related multimers Proc Natl Acad Sci U S A. 2018 Jan 23;115(4):798-803
Primary 409 cells 100 µM 72 hours To evaluate the effect of Miglustat on GD2 expression, results showed a significant decrease in GD2 expression Int J Mol Sci. 2023 Jun 8;24(12):9905
SF8628 cells 100 µM 72 hours To evaluate the effect of Miglustat on GD2 expression, results showed a significant decrease in GD2 expression Int J Mol Sci. 2023 Jun 8;24(12):9905
Neonatal rat cardiac fibroblasts (NRCFs) 100 μM and 200 μM 24 hours To evaluate the effect of Miglustat on ISO-induced cardiac fibroblast activation. Results showed that Miglustat significantly inhibited ISO-induced NRCFs proliferation, ECM deposition, and transdifferentiation. Mol Med. 2025 Feb 11;31(1):55

In Vivo:

Species
Animal Model
Administration Dosage Frequency Description References
C57BL/6J mice ISO-induced cardiac fibrosis model Oral 150 mg/kg and 300 mg/kg Twice daily for 7 days To evaluate the effect of Miglustat on ISO-induced cardiac dysfunction and fibrosis. Results showed that Miglustat significantly improved cardiac function, reduced fibrosis area, and suppressed the expression of fibrosis markers. Mol Med. 2025 Feb 11;31(1):55
NOD mice Experimental autoimmune encephalomyelitis (EAE) Oral 600 mg/kg Once daily for 2 weeks To study the therapeutic effect of Miglustat on EAE, results showed that Miglustat suppressed EAE progression, reduced axonal loss and demyelination, and decreased the recruitment of pro-inflammatory monocytes. Cell. 2019 Dec 12;179(7):1483-1498. e22
Mouse Mcoln1−/− mice Intraperitoneal injection 300 mg/kg body weight Daily from P10-P21, then three times a week until the end of the study Miglustat treatment delays motor deficits, cerebellar microgliosis, and Purkinje cell loss in Mcoln1?/? mice Neurobiol Dis. 2017 Sep;105:257-270

Clinical Trial:

NCT Number Conditions Phases Recruitment Completion Date Locations
NCT01760564 Niemann-Pick Disease Type C PHASE3 COMPLETED 2025-12-10 National Taiwan University Hos... More >>pital, Taipei, Taiwan Less <<
NCT03910621 Niemann-Pick Disease, Type C PHASE4 COMPLETED 2022-03-25 Peking University first Hospit... More >>al, Beijing, 100034, China|Xin Hua Hospital, Shanghai Jiao Tong University, Shanghai, 200092, China Less <<
NCT00319046 Gaucher Disease Type 1 PHASE3 COMPLETED 2010-07-01 -
NCT03822013 GM2 Gangliosidosis|Supportive ... More >>Care Less << PHASE3 RECRUITING 2024-12-30 Kashan University Of Medical S... More >>ciences, Kashan, Isfahan, Iran, Islamic Republic of|Mashhad University Of Medical Sciences, Mashhad, Khorasan, Iran, Islamic Republic of|Tehran University Of Medical Sciences, Tehran, Iran, Islamic Republic of Less <<

Protocol

Bio Calculators
Preparing Stock Solutions 1mg 5mg 10mg

1 mM

5 mM

10 mM

3.91mL

0.78mL

0.39mL

19.55mL

3.91mL

1.96mL

39.10mL

7.82mL

3.91mL

Dissolving Methods
Please choose the appropriate dissolution scheme according to your animal administration guide.For the following dissolution schemes, clear stock solution should be prepared according to in vitro experiments, and then cosolvent should be added in turn:

in order to ensure the reliability of the experimental results, the clarified stock solution can be properly preserved according to the storage conditions; The working fluid for in vivo experiment is recommended to be prepared now and used on the same day;

The percentage shown in front of the following solvent refers to the volume ratio of the solvent in the final solution; If precipitation or precipitation occurs in the preparation process, it can be assisted by heating and/or ultrasound.
Protocol 1
Protocol 2
The prepared working fluid is recommended to be prepared now and used up as soon as possible in a short period of time. The percentage shown in front of the following solvent refers to the volume ratio of the solvent in the final solution; If precipitation or precipitation occurs in the preparation process, it can be assisted by heating and/or ultrasound.
Protocol 1

References

 

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