Page last updated: 2024-09-27

isofagomine

Cross-References

ID SourceID
PubMed CID447607
CHEMBL ID206468
CHEBI ID194390
SCHEMBL ID581577
MeSH IDM0258559

Synonyms (35)

Synonym
IFM ,
5-hydroxymethyl-3,4-dihydroxypiperidine
(3r,4r,5r)-5-(hydroxymethyl)piperidine-3,4-diol
isofagomine ,
DB04545
afegostat
1OIF
CHEMBL206468 ,
AKOS006348872
(3r,4r,5r)-5-(hydroxymethyl)piperidine-3,4-diol, 8
d-isofagomine
bdbm50182801
CHEBI:194390
169105-89-9
afegostat (usan/inn)
D09576
afegostat [usan:inn]
g23ap190ys ,
iso-fagomine
3,4-piperidinediol, 5-(hydroxymethyl)-, (3r,4r,5r)-
5-hydroxymethyl-3,4-piperidinediol
unii-g23ap190ys
gtpl7410
SCHEMBL581577
isofagomine [mi]
afegostat [who-dd]
afegostat [usan]
afegostat [inn]
QPYJXFZUIJOGNX-HSUXUTPPSA-N
DTXSID20168651
isofagomine, hydrochloride
afegostat; isofagomine; (3r,4r,5r)-5-(hydroxymethyl)piperidine-3,4-diol
HY-14829
CS-0003581
Q21396373

Research Excerpts

Overview

ExcerptReference
"Isofagomine (IFG) is an acid β-glucosidase (GCase) active site inhibitor that acts as a pharmacological chaperone. "( Grabowski, GA; Hamler, R; Liou, B; Quinn, B; Setchell, KD; Sun, Y; Xu, YH; Zhang, W, 2012)

Drug Classes (1)

ClassDescription
piperidines
[compound class information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Protein Targets (16)

Potency Measurements

ProteinTaxonomyMeasurementAverage (µ)Min (ref.)Avg (ref.)Max (ref.)Bioassay(s)
glucocerebrosidaseHomo sapiens (human)Potency0.45030.01268.156944.6684AID2587; AID2588; AID2589; AID2590; AID2592; AID2595; AID2596; AID2597; AID2613; AID2671; AID488845; AID488849; AID488850; AID488851; AID488852; AID488853; AID488854
Fatty-acid amide hydrolase 1Rattus norvegicus (Norway rat)Potency0.63100.631016.842044.6684AID2596
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Inhibition Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Glycogen phosphorylase, muscle formOryctolagus cuniculus (rabbit)Ki5.20000.02504.89039.0000AID270952
Lysosomal acid glucosylceramidaseHomo sapiens (human)IC50 (µMol)0.05550.03002.35898.8000AID1884450; AID342801; AID500310; AID613052
Glycogen phosphorylase, liver formHomo sapiens (human)IC50 (µMol)0.68500.08201.16594.8000AID270950; AID342814
Glycogen phosphorylase, liver formRattus norvegicus (Norway rat)IC50 (µMol)0.68000.68000.92671.1000AID270949
Translocator proteinRattus norvegicus (Norway rat)Ki5.20000.00010.65108.9300AID270952
Sucrase-isomaltase, intestinalRattus norvegicus (Norway rat)IC50 (µMol)410.50000.04001.848310.0000AID613047; AID613048
Beta-glucosidaseAgrobacterium tumefaciensKi0.00700.00700.00700.0070AID263086
Glycogen debranching enzymeOryctolagus cuniculus (rabbit)IC50 (µMol)480.00000.11000.69752.1000AID342805
Trehalose synthase/amylase TreSMycobacterium tuberculosis CDC1551Ki140.00000.25001.37502.5000AID1799775
Lactase-phlorizin hydrolase Rattus norvegicus (Norway rat)IC50 (µMol)0.12000.12002.98674.4000AID613041
Lysosomal acid glucosylceramidaseBos taurus (cattle)IC50 (µMol)31.00005.60005.60005.6000AID613051
Beta-galactosidaseBos taurus (cattle)IC50 (µMol)3.60001.50002.70003.6000AID613054
Non-lysosomal glucosylceramidaseHomo sapiens (human)IC50 (µMol)0.02000.00030.08970.3000AID1408921
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Other Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Lysosomal acid glucosylceramidaseHomo sapiens (human)INH0.11000.11000.11000.1100AID617953
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Biological Processes (72)

Processvia Protein(s)Taxonomy
mitochondrion organizationLysosomal acid glucosylceramidaseHomo sapiens (human)
neuron projection developmentLysosomal acid glucosylceramidaseHomo sapiens (human)
glucosylceramide catabolic processLysosomal acid glucosylceramidaseHomo sapiens (human)
autophagyLysosomal acid glucosylceramidaseHomo sapiens (human)
lysosome organizationLysosomal acid glucosylceramidaseHomo sapiens (human)
cholesterol metabolic processLysosomal acid glucosylceramidaseHomo sapiens (human)
determination of adult lifespanLysosomal acid glucosylceramidaseHomo sapiens (human)
cellular response to starvationLysosomal acid glucosylceramidaseHomo sapiens (human)
response to pHLysosomal acid glucosylceramidaseHomo sapiens (human)
microglia differentiationLysosomal acid glucosylceramidaseHomo sapiens (human)
regulation of macroautophagyLysosomal acid glucosylceramidaseHomo sapiens (human)
antigen processing and presentationLysosomal acid glucosylceramidaseHomo sapiens (human)
lipid storageLysosomal acid glucosylceramidaseHomo sapiens (human)
cerebellar Purkinje cell layer formationLysosomal acid glucosylceramidaseHomo sapiens (human)
pyramidal neuron differentiationLysosomal acid glucosylceramidaseHomo sapiens (human)
respiratory electron transport chainLysosomal acid glucosylceramidaseHomo sapiens (human)
termination of signal transductionLysosomal acid glucosylceramidaseHomo sapiens (human)
lipid glycosylationLysosomal acid glucosylceramidaseHomo sapiens (human)
negative regulation of protein-containing complex assemblyLysosomal acid glucosylceramidaseHomo sapiens (human)
regulation of TOR signalingLysosomal acid glucosylceramidaseHomo sapiens (human)
positive regulation of proteasomal ubiquitin-dependent protein catabolic processLysosomal acid glucosylceramidaseHomo sapiens (human)
negative regulation of interleukin-6 productionLysosomal acid glucosylceramidaseHomo sapiens (human)
T cell differentiation in thymusLysosomal acid glucosylceramidaseHomo sapiens (human)
response to testosteroneLysosomal acid glucosylceramidaseHomo sapiens (human)
positive regulation of protein dephosphorylationLysosomal acid glucosylceramidaseHomo sapiens (human)
proteasome-mediated ubiquitin-dependent protein catabolic processLysosomal acid glucosylceramidaseHomo sapiens (human)
positive regulation of protein-containing complex disassemblyLysosomal acid glucosylceramidaseHomo sapiens (human)
negative regulation of MAP kinase activityLysosomal acid glucosylceramidaseHomo sapiens (human)
negative regulation of neuron apoptotic processLysosomal acid glucosylceramidaseHomo sapiens (human)
response to estrogenLysosomal acid glucosylceramidaseHomo sapiens (human)
sphingosine biosynthetic processLysosomal acid glucosylceramidaseHomo sapiens (human)
ceramide biosynthetic processLysosomal acid glucosylceramidaseHomo sapiens (human)
cell maturationLysosomal acid glucosylceramidaseHomo sapiens (human)
brain morphogenesisLysosomal acid glucosylceramidaseHomo sapiens (human)
homeostasis of number of cellsLysosomal acid glucosylceramidaseHomo sapiens (human)
negative regulation of inflammatory responseLysosomal acid glucosylceramidaseHomo sapiens (human)
neuromuscular processLysosomal acid glucosylceramidaseHomo sapiens (human)
neuron apoptotic processLysosomal acid glucosylceramidaseHomo sapiens (human)
establishment of skin barrierLysosomal acid glucosylceramidaseHomo sapiens (human)
microglial cell proliferationLysosomal acid glucosylceramidaseHomo sapiens (human)
motor behaviorLysosomal acid glucosylceramidaseHomo sapiens (human)
cellular response to tumor necrosis factorLysosomal acid glucosylceramidaseHomo sapiens (human)
hematopoietic stem cell proliferationLysosomal acid glucosylceramidaseHomo sapiens (human)
response to dexamethasoneLysosomal acid glucosylceramidaseHomo sapiens (human)
lymphocyte migrationLysosomal acid glucosylceramidaseHomo sapiens (human)
response to thyroid hormoneLysosomal acid glucosylceramidaseHomo sapiens (human)
beta-glucoside catabolic processLysosomal acid glucosylceramidaseHomo sapiens (human)
positive regulation of protein lipidationLysosomal acid glucosylceramidaseHomo sapiens (human)
positive regulation of neuronal action potentialLysosomal acid glucosylceramidaseHomo sapiens (human)
positive regulation of autophagy of mitochondrion in response to mitochondrial depolarizationLysosomal acid glucosylceramidaseHomo sapiens (human)
autophagosome organizationLysosomal acid glucosylceramidaseHomo sapiens (human)
regulation of lysosomal protein catabolic processLysosomal acid glucosylceramidaseHomo sapiens (human)
glycogen metabolic processGlycogen phosphorylase, liver formHomo sapiens (human)
5-phosphoribose 1-diphosphate biosynthetic processGlycogen phosphorylase, liver formHomo sapiens (human)
response to bacteriumGlycogen phosphorylase, liver formHomo sapiens (human)
glucose homeostasisGlycogen phosphorylase, liver formHomo sapiens (human)
necroptotic processGlycogen phosphorylase, liver formHomo sapiens (human)
glycogen catabolic processGlycogen phosphorylase, liver formHomo sapiens (human)
carbohydrate metabolic processBeta-galactosidaseHomo sapiens (human)
galactose catabolic processBeta-galactosidaseHomo sapiens (human)
heparan sulfate proteoglycan catabolic processBeta-galactosidaseHomo sapiens (human)
keratan sulfate catabolic processBeta-galactosidaseHomo sapiens (human)
glycosphingolipid catabolic processBeta-galactosidaseHomo sapiens (human)
response to cortisoneBeta-galactosidaseHomo sapiens (human)
response to Thyroglobulin triiodothyronineBeta-galactosidaseHomo sapiens (human)
glucosylceramide catabolic processLysosomal acid glucosylceramidaseBos taurus (cattle)
autophagyLysosomal acid glucosylceramidaseBos taurus (cattle)
lysosome organizationLysosomal acid glucosylceramidaseBos taurus (cattle)
cholesterol metabolic processLysosomal acid glucosylceramidaseBos taurus (cattle)
lipid glycosylationLysosomal acid glucosylceramidaseBos taurus (cattle)
regulation of TOR signalingLysosomal acid glucosylceramidaseBos taurus (cattle)
carbohydrate metabolic processBeta-galactosidaseBos taurus (cattle)
carbohydrate metabolic processNon-lysosomal glucosylceramidaseHomo sapiens (human)
glucosylceramide catabolic processNon-lysosomal glucosylceramidaseHomo sapiens (human)
cholesterol metabolic processNon-lysosomal glucosylceramidaseHomo sapiens (human)
bile acid metabolic processNon-lysosomal glucosylceramidaseHomo sapiens (human)
glycoside catabolic processNon-lysosomal glucosylceramidaseHomo sapiens (human)
central nervous system neuron developmentNon-lysosomal glucosylceramidaseHomo sapiens (human)
lipid glycosylationNon-lysosomal glucosylceramidaseHomo sapiens (human)
regulation of actin filament polymerizationNon-lysosomal glucosylceramidaseHomo sapiens (human)
regulation of microtubule polymerizationNon-lysosomal glucosylceramidaseHomo sapiens (human)
glycosphingolipid catabolic processNon-lysosomal glucosylceramidaseHomo sapiens (human)
regulation of membrane lipid distributionNon-lysosomal glucosylceramidaseHomo sapiens (human)
central nervous system developmentNon-lysosomal glucosylceramidaseHomo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Molecular Functions (22)

Processvia Protein(s)Taxonomy
galactosylceramidase activityLysosomal acid glucosylceramidaseHomo sapiens (human)
glucosylceramidase activityLysosomal acid glucosylceramidaseHomo sapiens (human)
signaling receptor bindingLysosomal acid glucosylceramidaseHomo sapiens (human)
scavenger receptor bindingLysosomal acid glucosylceramidaseHomo sapiens (human)
protein bindingLysosomal acid glucosylceramidaseHomo sapiens (human)
glucosyltransferase activityLysosomal acid glucosylceramidaseHomo sapiens (human)
steryl-beta-glucosidase activityLysosomal acid glucosylceramidaseHomo sapiens (human)
purine nucleobase bindingGlycogen phosphorylase, liver formHomo sapiens (human)
protein bindingGlycogen phosphorylase, liver formHomo sapiens (human)
ATP bindingGlycogen phosphorylase, liver formHomo sapiens (human)
glucose bindingGlycogen phosphorylase, liver formHomo sapiens (human)
glycogen phosphorylase activityGlycogen phosphorylase, liver formHomo sapiens (human)
AMP bindingGlycogen phosphorylase, liver formHomo sapiens (human)
vitamin bindingGlycogen phosphorylase, liver formHomo sapiens (human)
bile acid bindingGlycogen phosphorylase, liver formHomo sapiens (human)
identical protein bindingGlycogen phosphorylase, liver formHomo sapiens (human)
linear malto-oligosaccharide phosphorylase activityGlycogen phosphorylase, liver formHomo sapiens (human)
SHG alpha-glucan phosphorylase activityGlycogen phosphorylase, liver formHomo sapiens (human)
pyridoxal phosphate bindingGlycogen phosphorylase, liver formHomo sapiens (human)
beta-galactosidase activityBeta-galactosidaseHomo sapiens (human)
protein bindingBeta-galactosidaseHomo sapiens (human)
galactoside bindingBeta-galactosidaseHomo sapiens (human)
protein homodimerization activityBeta-galactosidaseHomo sapiens (human)
galactosylceramidase activityLysosomal acid glucosylceramidaseBos taurus (cattle)
glucosylceramidase activityLysosomal acid glucosylceramidaseBos taurus (cattle)
glucosyltransferase activityLysosomal acid glucosylceramidaseBos taurus (cattle)
steryl-beta-glucosidase activityLysosomal acid glucosylceramidaseBos taurus (cattle)
beta-galactosidase activityBeta-galactosidaseBos taurus (cattle)
galactosylceramidase activityNon-lysosomal glucosylceramidaseHomo sapiens (human)
glucosylceramidase activityNon-lysosomal glucosylceramidaseHomo sapiens (human)
beta-glucosidase activityNon-lysosomal glucosylceramidaseHomo sapiens (human)
glucosyltransferase activityNon-lysosomal glucosylceramidaseHomo sapiens (human)
steryl-beta-glucosidase activityNon-lysosomal glucosylceramidaseHomo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Ceullar Components (21)

Processvia Protein(s)Taxonomy
lysosomeLysosomal acid glucosylceramidaseHomo sapiens (human)
lysosomal membraneLysosomal acid glucosylceramidaseHomo sapiens (human)
endoplasmic reticulumLysosomal acid glucosylceramidaseHomo sapiens (human)
Golgi apparatusLysosomal acid glucosylceramidaseHomo sapiens (human)
trans-Golgi networkLysosomal acid glucosylceramidaseHomo sapiens (human)
lysosomal lumenLysosomal acid glucosylceramidaseHomo sapiens (human)
extracellular exosomeLysosomal acid glucosylceramidaseHomo sapiens (human)
extracellular regionGlycogen phosphorylase, liver formHomo sapiens (human)
cytosolGlycogen phosphorylase, liver formHomo sapiens (human)
secretory granule lumenGlycogen phosphorylase, liver formHomo sapiens (human)
extracellular exosomeGlycogen phosphorylase, liver formHomo sapiens (human)
ficolin-1-rich granule lumenGlycogen phosphorylase, liver formHomo sapiens (human)
cytoplasmGlycogen phosphorylase, liver formHomo sapiens (human)
extracellular regionBeta-galactosidaseHomo sapiens (human)
cytoplasmBeta-galactosidaseHomo sapiens (human)
Golgi apparatusBeta-galactosidaseHomo sapiens (human)
azurophil granule lumenBeta-galactosidaseHomo sapiens (human)
lysosomal lumenBeta-galactosidaseHomo sapiens (human)
intracellular membrane-bounded organelleBeta-galactosidaseHomo sapiens (human)
perinuclear region of cytoplasmBeta-galactosidaseHomo sapiens (human)
extracellular exosomeBeta-galactosidaseHomo sapiens (human)
ficolin-1-rich granule lumenBeta-galactosidaseHomo sapiens (human)
vacuoleBeta-galactosidaseHomo sapiens (human)
lysosomeLysosomal acid glucosylceramidaseBos taurus (cattle)
lysosomal membraneLysosomal acid glucosylceramidaseBos taurus (cattle)
endoplasmic reticulumLysosomal acid glucosylceramidaseBos taurus (cattle)
Golgi apparatusLysosomal acid glucosylceramidaseBos taurus (cattle)
trans-Golgi networkLysosomal acid glucosylceramidaseBos taurus (cattle)
lysosomeBeta-galactosidaseBos taurus (cattle)
Golgi membraneNon-lysosomal glucosylceramidaseHomo sapiens (human)
endoplasmic reticulum membraneNon-lysosomal glucosylceramidaseHomo sapiens (human)
smooth endoplasmic reticulumNon-lysosomal glucosylceramidaseHomo sapiens (human)
cytosolNon-lysosomal glucosylceramidaseHomo sapiens (human)
plasma membraneNon-lysosomal glucosylceramidaseHomo sapiens (human)
membraneNon-lysosomal glucosylceramidaseHomo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (87)

Assay IDTitleYearJournalArticle
AID577516Binding affinity to recombinant Gcase assessed as change in melting temperature at 500 uM by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID577335Increase of GCase N370S mutant trafficking to lysosomes in human fibroblast cells assessed as colocalization of Gcase with Lamp2 at 1 uM after 5 days by immunofluorescence method2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID1408923Chaperone activity at beta-galactosidase p.H281Y mutant in Zurich2 fibroblasts derived from GM1-gangliosidosis patient assessed as increase in enzyme activity using 4-methylumbelliferyl beta-D-galactopyranoside as substrate preincubated for 2 days followe2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations.
AID342972Inhibition of basal glycogen degradation in hepatocytes2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID1601617Activation of lysosomal beta-glucocerebrosidase N370S mutant in fibroblast cells derived from Gaucher's disease patient at 5 uM using PFB-FDGlu as substrate preincubated for 3 days followed by substrate addition and measured after 1 hr for every 20 mins i2019Journal of medicinal chemistry, 02-14, Volume: 62, Issue:3
Conversion of Quinazoline Modulators from Inhibitors to Activators of β-Glucocerebrosidase.
AID1408921Inhibition of beta-glucosidase derived from human peripheral blood mononuclear cell lysate using 4-methylumbelliferyl beta-D-glucopyranoside as substrate after 2 hrs by fluorescence analysis2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations.
AID270951Binding affinity to non-phosphorylated form of rabbit muscle glycogen phosphorylase at 50 mM in absence of phosphate2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Iminosugars as potential inhibitors of glycogenolysis: structural insights into the molecular basis of glycogen phosphorylase inhibition.
AID270948Inhibition of phosphorylated form of rabbit muscle glycogen phosphorylase2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Iminosugars as potential inhibitors of glycogenolysis: structural insights into the molecular basis of glycogen phosphorylase inhibition.
AID613050Inhibition of rat intestinal cellobiase assessed as production of p-nitrophenol by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID500310Inhibition of GCase assessed as 4-methylumbelliferone release assay after 30 mins by fluorimetry2007Nature chemical biology, Feb, Volume: 3, Issue:2
Structure of acid beta-glucosidase with pharmacological chaperone provides insight into Gaucher disease.
AID620279Inhibition of sweet almond beta-glucosidase2011Bioorganic & medicinal chemistry, Oct-01, Volume: 19, Issue:19
Synthesis of new six- and seven-membered 1-N-iminosugars as promising glycosidase inhibitors.
AID342800Inhibition of human lysosomal alpha-glucosidase at 1000 uM2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID613054Inhibition of bovine liver beta-galactosidase assessed as production of p-nitrophenol by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID731610Induction of beta glucosidase L444P mutant activity in fibroblasts derived from Gaucher disease patient using 4MUG as substrate relative to control2013Journal of medicinal chemistry, Apr-11, Volume: 56, Issue:7
Pharmacological chaperones as therapeutics for lysosomal storage diseases.
AID577524Binding affinity to recombinant Gcase assessed as change in melting temperature at 1 uM by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID1321666Activation of GCase N370S mutant in human Gaucher disease patient fibroblasts assessed as increase in GCase protein levels at 20 and 50 uM measured after 3 days by immunoblot analysis2016Journal of medicinal chemistry, Sep-22, Volume: 59, Issue:18
Design and Synthesis of Potent Quinazolines as Selective β-Glucocerebrosidase Modulators.
AID270952Inhibition of non-phosphorylated form of rabbit muscle glycogen phosphorylase in presence of phosphate2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Iminosugars as potential inhibitors of glycogenolysis: structural insights into the molecular basis of glycogen phosphorylase inhibition.
AID613046Inhibition of rat intestinal maltase assessed as production of D-glucose using maltose as substrate after 10 to 30 mins by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID731607Induction of beta glucosidase L444P mutant in mouse brain administered with drinking water measured after 4 weeks relative to control2013Journal of medicinal chemistry, Apr-11, Volume: 56, Issue:7
Pharmacological chaperones as therapeutics for lysosomal storage diseases.
AID613039Inhibition of coffee beans alpha-galactosidase assessed as production of p-nitrophenol at 1000 uM by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID342812Increase in lysosomal beta-glucosidase N370S mutant activity in human Gaucher GM00372 fibroblast at 10 uM after 4 days2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID342801Inhibition of human lysosomal beta-glucosidase2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID577514Inhibition of alpha-galactosidase2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID1125778Increase of intracellular activity of beta-glucocerebrosidase in human N370S cells using 4-methylumbelliferyl-beta-D-glucopyranoside as substrate at 50 uM after 4 days relative to control2014Bioorganic & medicinal chemistry, Apr-15, Volume: 22, Issue:8
Docking and SAR studies of calystegines: binding orientation and influence on pharmacological chaperone effects for Gaucher's disease.
AID1408920Inhibition of alpha-galactosidase derived from human peripheral blood mononuclear cell lysate using 4-methylumbelliferyl alpha-D-galactopyranoside as substrate after 2 hrs by fluorescence analysis2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations.
AID731621Induction of beta glucosidase N370S mutant activity in primary skin fibroblast derived from Gaucher disease patient at 30 uM after 5 days relative to control2013Journal of medicinal chemistry, Apr-11, Volume: 56, Issue:7
Pharmacological chaperones as therapeutics for lysosomal storage diseases.
AID613051Inhibition of bovine liver beta-glucosidase assessed as production of 4-methylumbelliferone using 4-methylumbelliferyl beta-D-glucoside as substrate by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID613041Inhibition of rat intestinal lactase assessed as production of p-nitrophenol by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID1321660Binding affinity to synthetic recombinant wild type GCase enzyme velaglucerase alfa (unknown origin) assessed as thermal stabilization measured as increase in melting temperature at pH 5 measured every 10 secs by Sypro Orange-dye based fluorescence therma2016Journal of medicinal chemistry, Sep-22, Volume: 59, Issue:18
Design and Synthesis of Potent Quinazolines as Selective β-Glucocerebrosidase Modulators.
AID500313Restoration of GCase N370S mutant trafficking to lysosomes in human DMN89.15 fibroblast cells assessed as colocalization of Gcase with Lamp1 at 100 uM after 5 days by immunofluorescence method2007Nature chemical biology, Feb, Volume: 3, Issue:2
Structure of acid beta-glucosidase with pharmacological chaperone provides insight into Gaucher disease.
AID617946Inhibition of alpha-glucosidase2011Bioorganic & medicinal chemistry, Sep-15, Volume: 19, Issue:18
Synthesis and glycosidase inhibitory profiles of functionalised morpholines and oxazepanes.
AID577520Binding affinity to recombinant Gcase assessed as change in melting temperature at 100 uM by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID577333Binding affinity to recombinant Gcase assessed as change in melting temperature by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID270950Inhibition of phosphorylated form of pig liver glycogen phosphorylase2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Iminosugars as potential inhibitors of glycogenolysis: structural insights into the molecular basis of glycogen phosphorylase inhibition.
AID617953Inhibition of beta-glucosidase2011Bioorganic & medicinal chemistry, Sep-15, Volume: 19, Issue:18
Synthesis and glycosidase inhibitory profiles of functionalised morpholines and oxazepanes.
AID1601619Activation of lysosomal beta-glucocerebrosidase activity in human healthy fibroblast cells at 5 uM using PFB-FDGlu as substrate preincubated for 3 days followed by substrate addition and measured after 1 hr for every 20 mins in presence of bafilomycin by 2019Journal of medicinal chemistry, 02-14, Volume: 62, Issue:3
Conversion of Quinazoline Modulators from Inhibitors to Activators of β-Glucocerebrosidase.
AID579819Activation of beta-glucocerebrosidase in human N370S cells assessed as decrease of residual activity at 0.05 to 100 uM after 3 days2011Bioorganic & medicinal chemistry letters, Mar-01, Volume: 21, Issue:5
Synthesis of N-alkylated noeurostegines and evaluation of their potential as treatment for Gaucher's disease.
AID577332Increase of GCase trafficking to lysosomes in human fibroblast cells assessed as colocalization of Gcase with Lamp2 at 10 uM after 5 days by immunofluorescence method2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID731611Induction of beta glucosidase N370S mutant activity in fibroblast derived from Gaucher disease patient at 10 to 50 uM relative to control2013Journal of medicinal chemistry, Apr-11, Volume: 56, Issue:7
Pharmacological chaperones as therapeutics for lysosomal storage diseases.
AID1412120Chaperone activity at GBA N370S mutant in Gaucher disease patient-derived fibroblasts assessed as restoration of enzyme activity at 1 to 10000 nM after 4 days by 4-methylumbelliferyl-beta-D-glucopyranoside substrate based fluorescence assay2017MedChemComm, Nov-01, Volume: 8, Issue:11
Orthoester functionalized
AID270958Inhibition of glucagon-stimulated glycogen degradation in hepatocytes2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Iminosugars as potential inhibitors of glycogenolysis: structural insights into the molecular basis of glycogen phosphorylase inhibition.
AID577525Binding affinity to recombinant Gcase assessed as change in melting temperature at 0.1 uM by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID731595Induction of beta glucosidase N370S mutant activity in fibroblasts derived from Gaucher disease type 1 patient at 30 uM relative to control2013Journal of medicinal chemistry, Apr-11, Volume: 56, Issue:7
Pharmacological chaperones as therapeutics for lysosomal storage diseases.
AID1601618Activation of lysosomal beta-glucocerebrosidase L444P mutant in fibroblast cells derived from Gaucher's disease patient at 5 uM using PFB-FDGlu as substrate preincubated for 3 days followed by substrate addition and measured after 1 hr for every 20 mins i2019Journal of medicinal chemistry, 02-14, Volume: 62, Issue:3
Conversion of Quinazoline Modulators from Inhibitors to Activators of β-Glucocerebrosidase.
AID613042Inhibition of jack beans alpha-mannosidase assessed as production of p-nitrophenol at 1000 uM by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID1884450Inhibition of human GCase assessed as reduction of 4-methylumbelliferone liberation using 4-methylumbelliferyl-beta-glucopyranoside as substrate preincubated with enzyme for 10 mins followed by substrate addition by fluorescence spectrophotometry2022European journal of medicinal chemistry, Aug-05, Volume: 238trans, trans-2-C-Aryl-3,4-dihydroxypyrrolidines as potent and selective β-glucosidase inhibitors: Pharmacological chaperones for Gaucher disease.
AID577521Binding affinity to recombinant Gcase assessed as change in melting temperature at 50 uM by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID613043Inhibition of snail beta-mannosidase assessed as production of p-nitrophenol at 1000 uM by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID1119120Inhibition of glucocerebrosidase N370S mutant in spleen homogenates of patient with Gaucher disease using resorufin-beta-D-glucopyranoside as substrate after 20 mins fluorescence assay2012MedChemComm, Jan, Volume: 3, Issue:1
Non-iminosugar glucocerebrosidase small molecule chaperones.
AID1408922Inhibition of beta-galactosidase derived from human peripheral blood mononuclear cell lysate using 4-methylumbelliferyl beta-D-galactopyranoside as substrate after 2 hrs by fluorescence analysis2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations.
AID497872Inhibition of wild type Bacteroides thetaiotaomicron beta-mannosidase 2A expressed in Escherichia coli BL21 (DE3) at 37 degC and Ph 5.6 by 2,4-dinitrophenolate release assay2008Nature chemical biology, May, Volume: 4, Issue:5
Structural and biochemical evidence for a boat-like transition state in beta-mannosidases.
AID342796Inhibition of rat intestinal brush border membrane isomaltase at 1000 uM2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID342805Inhibition of rabbit muscle amylo-1,6-glucosidase2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID577336Increase of GCase trafficking to lysosomes in human fibroblast cells assessed as colocalization of Gcase with Lamp2 at 1 uM after 5 days by immunofluorescence method2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID577519Binding affinity to recombinant Gcase assessed as change in melting temperature at 200 uM by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID500311Increase in Gcase N370S mutant activity in human DMN89.15 fibroblast cell lysate assessed as 4-methylumbelliferone release after 5 days by fluorimetry2007Nature chemical biology, Feb, Volume: 3, Issue:2
Structure of acid beta-glucosidase with pharmacological chaperone provides insight into Gaucher disease.
AID270949Inhibition of phosphorylated form of rat liver glycogen phosphorylase2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Iminosugars as potential inhibitors of glycogenolysis: structural insights into the molecular basis of glycogen phosphorylase inhibition.
AID613052Inhibition of human lysosome beta-glucosidase assessed as production of 4-methylumbelliferone using 4-methylumbelliferyl beta-D-glucoside as substrate by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID613047Inhibition of rat intestinal isomaltase assessed as production of p-nitrophenol at by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID342794Inhibition of rat intestinal brush border membrane maltase at 1000 uM2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID577518Binding affinity to recombinant Gcase assessed as change in melting temperature at 300 uM by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID270947Inhibition of non-phosphorylated form of rabbit muscle glycogen phosphorylase2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Iminosugars as potential inhibitors of glycogenolysis: structural insights into the molecular basis of glycogen phosphorylase inhibition.
AID342803Inhibition of rabbit glycogen phosphorylase B2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID342813Increase in lysosomal beta-glucosidase N370S mutant activity in human Gaucher GM00372 fibroblast at 500 uM after 4 days2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID577522Binding affinity to recombinant Gcase assessed as change in melting temperature at 25 uM by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID263086Inhibition of Agrobacterium sp. beta-glucosidase2006Bioorganic & medicinal chemistry letters, Apr-15, Volume: 16, Issue:8
Fluorescent glycosidase inhibiting 1,5-dideoxy-1,5-iminoalditols.
AID577517Binding affinity to recombinant Gcase assessed as change in melting temperature at 400 uM by fluorescence thermal shift technique2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID1601620Induction of post-ER GCase activity in fibroblast cells harboring N370S/84GG double mutant derived from Gaucher's disease patient at 25 uM using PFB-FDGlu as substrate preincubated for 3 days followed by substrate addition and measured after 1 hr for ever2019Journal of medicinal chemistry, 02-14, Volume: 62, Issue:3
Conversion of Quinazoline Modulators from Inhibitors to Activators of β-Glucocerebrosidase.
AID1408919Inhibition of alpha-glucosidase derived from human peripheral blood mononuclear cell lysate using 4-methylumbelliferyl alpha-D-glucopyranoside as substrate after 2 hrs by fluorescence analysis2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations.
AID613031Inhibition of rice alpha-glucosidase assessed as production of D-glucose using maltose as substrate at 1000 uM after 10 to 30 mins by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID613045Inhibition of Penicillium decumbens alpha-L-rhamnosidase assessed as production of p-nitrophenol at 1000 uM by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID577331Increase of GCase N370S mutant trafficking to lysosomes in human fibroblast cells assessed as colocalization of Gcase with Lamp2 at 10 uM after 5 days by immunofluorescence method2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID342973Inhibition of glucagon-stimulated glycogen degradation in hepatocytes2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID613044Inhibition of bovine epididymis alpha-L-fucosidase assessed as production of p-nitrophenol at 1000 uM by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID577513Inhibition of alpha-glucosidase2011Journal of medicinal chemistry, Feb-24, Volume: 54, Issue:4
Evaluation of quinazoline analogues as glucocerebrosidase inhibitors with chaperone activity.
AID500312Induction of Gcase N370S mutant distribution in human DMN89.15 fibroblast cells at 100 uM after 5 days by antibody staining based laser scanning confocal microscopy2007Nature chemical biology, Feb, Volume: 3, Issue:2
Structure of acid beta-glucosidase with pharmacological chaperone provides insight into Gaucher disease.
AID342798Inhibition of rat intestinal brush border membrane sucrase at 1000 uM2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID613048Inhibition of rat intestinal sucrase assessed as production of p-nitrophenol at by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID1119122Induction of glucocerebrosidase N370S mutant translocation in fibroblasts of patient with Gaucher disease assessed as co-localization of LAMP-1 in lysosomes at 1 uM after 5 days by laser scanning confocal microscopic analysis2012MedChemComm, Jan, Volume: 3, Issue:1
Non-iminosugar glucocerebrosidase small molecule chaperones.
AID342814Inhibition of liver glycogen phosphorylase A2008Bioorganic & medicinal chemistry, Aug-01, Volume: 16, Issue:15
In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell cultures.
AID270957Inhibition of glycogen degradation in hepatocytes2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Iminosugars as potential inhibitors of glycogenolysis: structural insights into the molecular basis of glycogen phosphorylase inhibition.
AID1408924Cytotoxicity against human GM02456 fibroblasts after 5 days by MTT assay2018Bioorganic & medicinal chemistry, 11-01, Volume: 26, Issue:20
4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations.
AID613049Inhibition of almond beta-glucosidase assessed as production of 4-methylumbelliferone using 4-methylumbelliferyl beta-D-glucoside as substrate by spectrophotometry2011Bioorganic & medicinal chemistry, Jun-01, Volume: 19, Issue:11
Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors.
AID731609Induction of beta glucosidase N370S mutant activity in fibroblast derived from Gaucher disease patient at 10 uM relative to control2013Journal of medicinal chemistry, Apr-11, Volume: 56, Issue:7
Pharmacological chaperones as therapeutics for lysosomal storage diseases.
AID1601621Induction of post-ER GCase level in fibroblast cells harboring N370S/84GG double mutant derived from Gaucher's disease patient at 25 uM by immunoblotting analysis2019Journal of medicinal chemistry, 02-14, Volume: 62, Issue:3
Conversion of Quinazoline Modulators from Inhibitors to Activators of β-Glucocerebrosidase.
AID1799390Btman2A from Article 10.1038/nchembio.81: \\Structural and biochemical evidence for a boat-like transition state in beta-mannosidases.\\2008Nature chemical biology, May, Volume: 4, Issue:5
Structural and biochemical evidence for a boat-like transition state in beta-mannosidases.
AID1799775Inhibition Assay from Article 10.1074/jbc.M111.280362: \\Mechanistic analysis of trehalose synthase from Mycobacterium smegmatis.\\2011The Journal of biological chemistry, Oct-14, Volume: 286, Issue:41
Mechanistic analysis of trehalose synthase from Mycobacterium smegmatis.
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (94)

TimeframeStudies, This Drug (%)All Drugs %
pre-19900 (0.00)18.7374
1990's3 (3.19)18.2507
2000's41 (43.62)29.6817
2010's46 (48.94)24.3611
2020's4 (4.26)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials0 (0.00%)5.53%
Reviews3 (3.16%)6.00%
Case Studies0 (0.00%)4.05%
Observational0 (0.00%)0.25%
Other92 (96.84%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]