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Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!2025-02-06
20 Results for "
Fabry
" in MedChemExpress (MCE) Product Catalog:
Products (20)
20 Results for "Fabry" in MCE Product Catalog:
3
3 Cited Publications
Cat. No.: HY-16743
CAS No.: 1401090-53-6
Synonyms: Venglustat; SAR402671; GZ402671
Target:
Research Areas:
Ibiglustat (Venglustat) is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat can be used for the research of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease .
3
3 Cited Publications
Cat. No.: HY-16743A
CAS No.: 1629063-78-0
Synonyms: Venglustat (L-Malic acid); SAR402671 (L-Malic acid); GZ402671 (L-Malic acid)
Target:
Research Areas:
Ibiglustat (Venglustat) L-Malic acid is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat L-Malic acid can be used for the research of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease .
3
3 Cited Publications
Cat. No.: HY-16743B
CAS No.: 1629063-80-4
Synonyms: Venglustat succinate; SAR402671 succinate; GZ402671 succinate
Target:
Research Areas:
Ibiglustat (Venglustat) succinate is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat succinate can be used for the research of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease .
2
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Migalastat (GR181413A free base) hydrochloride is an orally active α-galactosidase A molecular chaperone, with an IC50 value of 0.04 μM for human α-Gal A. Migalastat binds to the active site of certain unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. After dissociation in the acidic environment, Migalastat enables the mutant α-galactosidase A to exhibit biological activity .
Synonyms: NB-DGJ; N-(n-Butyl)deoxygalactonojirimycin
Target:
Research Areas:
Lucerastat, the galactose form of Miglustat, is an orally-available inhibitor of glucosylceramide synthase (GCS). Lucerastat has the potential for Fabry disease study .
Cat. No.: HY-N12408
CAS No.: 126550-86-5
Synonyms: Lyso-Gb3; Lyso-GL3
Target:
Synonyms: 4-Methylumbelliferyl-α-D-Glucose; 4-Methylumbelliferyl-α-D-Glucoside; 4-MU-α-D-Glucopyranoside
Target:
Research Areas:
4-Methylumbelliferyl-α-D-Glucopyranoside (4-Methylumbelliferyl-α-D-Glucose) is a fluorescent substrate for α-glucosidase, which releases the fluorescent moiety 4-methylumbelliferyl (4-MU) upon cleavage. 4-MU has pH-dependent fluorescence excitation activity, with excitation wavelengths of 320 nm at low pH (1.97-6.72) and 360 nm at high pH (7.12-10.3), respectively. The emission wavelength of 4-Methylumbelliferyl-α-D-Glucopyranoside increases with decreasing pH, ranging from 445-455 nm. 4-Methylumbelliferyl-α-D-Glucopyranoside can be used as a biomarker for Fabry and Pompe diseases to quantify α-glucosidase activity in infant blood spot samples.
Cat. No.: HY-156209
CAS No.: 137896-85-6
Target:
Research Areas:
Gb3 (d18:1/16:0) (Compound 1) is the P k antigen of the P blood group system. Gb3 (d18:1/16:0) acts as a cell surface receptor. Gb3 (d18:1/16:0) is used for research on Burkitt's lymphoma, teratocarcinoma, embryonal carcinoma, and Fabry's disease .
Cat. No.: HY-156155
CAS No.: 2839916-15-1
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Research Areas:
GYS1-IN-1 (compound 783) is a glycogen synthase 1(GYS1) inhibitor. GYS1-IN-1 can be used for the study of GYS1 -mediated disease, such as Gaucher and Fabry diseases .
Cat. No.: HY-172552
CAS No.: 3038234-95-3
Target:
Research Areas:
RECTAS-2.0 is a small molecule designed to correct RNA mis-splicing caused by the GLA c.639+919G>A mutation, used for the research of Fabry disease .
Cat. No.: HY-14929
CAS No.: 108147-54-2
Synonyms: GR181413A free base; 1-Deoxygalactonojirimycin
Target:
Research Areas:
Migalastat (GR181413A free base) is an orally active α-galactosidase A molecular chaperone, with an IC50 value of 0.04 μM for human α-Gal A. Migalastat binds to the active site of certain unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. After dissociation in the acidic environment, Migalastat enables the mutant α-galactosidase A to exhibit biological activity .
Cat. No.: HY-172546
Synonyms: LysoGM2 ammonium
Target:
Research Areas:
Lyso-monosialoganglioside GM2 (LysoGM2) ammonium is a lysosphingolipid and protein kinase C inhibitor (IC50: 50 μM). Lyso-monosialoganglioside GM2 ammonium can inhibit the binding of protein kinase C to Phorbol 12,13-dibutyrate (HY-18985). Lyso-monosialoganglioside GM2 ammonium is a specific biomarker for GM2 gangliosidosis. Lyso-monosialoganglioside GM2 ammonium can be used in the research of sphingolipidoses .
Cat. No.: HY-156209S
CAS No.: 2738376-84-4
Cat. No.: HY-106392R
CAS No.: 141206-42-0
Synonyms: NB-DGJ (Standard); N-(n-Butyl)deoxygalactonojirimycin (Standard)
Research Areas:
Lucerastat (Standard) is the analytical standard of Lucerastat. This product is intended for research and analytical applications. Lucerastat, the galactose form of Miglustat, is an orally-available inhibitor of glucosylceramide synthase (GCS). Lucerastat has the potential for Fabry disease study .
Synonyms: Ceramide trihexoside (d18:1/17:0); Gb3(d18:1)(C17:0)
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Research Areas:
C17 Globotriaosylceramide (d18:1/17:0) is a sphygosine lipid that can be used to quantify Globotriaosylceramide in plasma and urine of patients with Fabry disease by gas chromatography-mass spectrometry (GC-MS) .
Cat. No.: HY-16743C
CAS No.: 1629063-79-1
Synonyms: Venglustat hydrochloride; SAR402671 hydrochloride; GZ402671 hydrochloride
Target:
Research Areas:
Ibiglustat hydrochloride is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat hydrochloride can be used for the research of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease .
Cat. No.: HY-156208
CAS No.: 574738-16-2
Synonyms: AdaGalCer(d18:1/2:0); Admantanyl galactosylceramide (d18:1/2:0); Admantanyl galCer(d18:1/2:0)
Research Areas:
C2 Adamantanyl galactosylceramide (AdaGalCer) (d18:1/2:0) is a bioactive sphingolipid. C2 Adamantanyl galactosylceramide (d18:1/2:0) stimulates glucocerebrosidase activity in vitro. C2 Adamantanyl galactosylceramide (d18:1/2:0) inhibits microsomal LacCer and Gb3 synthase, and inhibits cell sulfatide synthesis. C2 Adamantanyl galactosylceramide (d18:1/2:0) reduces glucosylceramide (GlcCer) levels in normal and lysosomal storage disease (LSD) cells. C2 Adamantanyl galactosylceramide (d18:1/2:0) acts as a substrate for A4GALT and is able to lower Gb3 levels with an IC50 concentration of 40 μM in fabry disease cells .
Cat. No.: HY-N11157
CAS No.: 82005-99-0
Category:
Target:
Cat. No.: HY-175076
Target:
Research Areas:
Globotriaosylceramide (non-hydroxy) (porcine RBC) is a glycosphingolipid found in the cell membranes of mammals. Globotriaosylceramide (non-hydroxy) (porcine RBC) is the core pathogenic molecule of Fabry disease, and its level can be downregulated by supplementing α-galactosidase A. Globotriaosylceramide (non-hydroxy) (porcine RBC) amplifies B-cell receptor signals by regulating the localization of co-receptor CD19, and can be used as a vaccine adjuvant, capable of guiding the generation of broad-spectrum and cross-protective antibody responses. Globotriaosylceramide (non-hydroxy) (porcine RBC) promotes wound healing and reduces scar formation by regulating fibroblast heterogeneity .
Cat. No.: HY-181469
Synonyms: Adamantanyl GluCer (d18:1/2:0); Adamantanyl glucosylceramide (d18:1/2:0); AdaGluCer (d18:1/2:0)
Target:
Research Areas:
C2 Adamantanyl glucosylceramide (d18:1/2:0) (Adamantanyl GluCer (d18:1/2:0)) is an inhibitor of glucocerebrosidase (GCC) and LacCer synthase. At low doses, C2 Adamantanyl glucosylceramide (d18:1/2:0) increases intracellular glycolipid levels by inhibiting glucocerebrosidase. C2 Adamantanyl glucosylceramide (d18:1/2:0) alters glycolipid metabolism. C2 Adamantanyl glucosylceramide (d18:1/2:0) can be used for the research of Gaucher disease and Fabry disease .
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Protocols
Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!A suitable model is crucial in drug screening experiments. Organs can mimic the three-dimensional functional structure of internal organs, have similar spatial organization to corresponding organs, maintain some key characteristics, and reproduce some physiological functions. They are widely used for modeling and personalized drug screening physiological functions.2025-02-06
