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T8986 |
ML198
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Glucokinase
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ML198 is a novel activator and non-inhibitory chaperone of glucocerebrosidase. ML198 serves as a starting point to develop a novel approach towards small molecule treatment for patients suffering from Gaucher disease. |
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T0920 |
Ambroxol
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Ambroxolum,NA-872 |
P450
,
Autophagy
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Ambroxol (NA-872) is a secretolytic agent used in the treatment of respiratory diseases associated with viscid or excessive mucus. It is the active ingredient of Mucosolvan, Lasolvan or Mucoangin. The substance is a muco... |
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T6234 |
Ambroxol hydrochloride
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Mucosolvan,Mucoangin,Ambroxol HCl |
Sodium Channel
,
Autophagy
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Ambroxol hydrochloride (Mucosolvan) is a metabolite of BROMHEXINE that stimulates mucociliary action and clears the air passages in the respiratory tract. It is usually administered as the hydrochloride. |
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TQ0155 |
Miglustat hydrochloride
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NB-DNJ hydrochloride,N-Butyldeoxynojirimycin hydrochloride,OGT918 hydrochloride |
Transferase
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T3663 |
Eliglustat
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GENZ-112638,Genz 99067 |
Transferase
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Eliglustat (Genz 99067) is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease. |
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T40544 |
NCGC00092410
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ML008 |
Glucosidase
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NCGC00092410 (ML008) is a glucocerebrosidase (GC) inhibitor with IC50 value of 31 nM. It can be used for the study of Gaucher disease caused by mutation of glucocerebrase gene. |
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T8990 |
ML266
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Glucokinase
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ML266, a glucocerebrosidase (GCase) chaperone with an IC50 of 2.5 µM, facilitates the transport of the mutant protein to the lysosome, thereby restoring GCase activity without inhibiting the enzyme's function. This compo... |
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T39105 |
Ibiglustat succinate
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GZ402671succinate,Venglustat succinate,Ibiglustat succinate,SAR402671succinate |
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Ibiglustat (Venglustat) succinate is an orally active, brain-penetrant inhibitor of glucosylceramide synthase (GCS). It is utilized in the investigation of Gaucher disease type 3, Parkinson's disease associated with GBA ... |
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T73716 |
Miglustat-d9 HCl
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Miglustat-d 9 (hydrochloride), a deuterium-labeled variant of Miglustat (hydrochloride), functions as a glucosylceramide synthase inhibitor. Its primary application is in the treatment of Type I Gaucher disease (GD1) [1]... |
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T37440 |
C2 Adamantanyl Galactosylceramide (d18:1/2:0)
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C2 Adamantanyl Galactosylceramide (d18:1/2:0) (AdaGalCer) is a bioactive sphingolipid. It reduces globotriaosylceramide 3 synthesis from exogenous lactosylceramide in microsomes. AdaGalCer stimulates recombinant glucocer... |
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T80994 |
Therapeutic agent-1
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Therapeutic Agent-1, a heteroaryl compound, serves as an enzyme replacement therapy to enhance glucocerebrosidase activity in the treatment of Gaucher disease [1]. |
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T36582 |
Glucocerebrosides (Gaucher's spleen)
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Glucocerebrosides are formed by the tethering of glucose to a ceramide by glucosylceramide synthase. They are present in neuronal and non-neuronal mammalian tissues and are found at low quantities in a large number of pl... |
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T78671 |
Glucocerebrosidase-IN-1 hydrochloride
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Glucosidase
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Glucocerebrosidase-IN-1 (compound 11a) hydrochloride is a potent, selective GCase (glucocerebrosidase) inhibitor with IC50 and Ki values of 29.3 μM and 18.5 μM, respectively. It is utilized in the research of Gaucher dis... |
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T60353 |
Glucocerebrosidase-IN-1
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Glucocerebrosidase-IN-1 (compound 11a) is a potent and selective GCase (glucocerebrosidase) inhibitor, with an IC 50 of 29.3 μM and a K i of 18.5 μM. Due to GCase involvement in pathological disorders consequent to enzym... |
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T62898 |
Glucosylceramide synthase-IN-1
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Glucosylceramide synthase-IN-1 (T-036) is a potent, orally active, blood-brain barrier permeable glucosylceramide synthase (GCS) inhibitor that acts on human GCS (IC50: 31 nM) and mouse GCS (IC50: 51 nM). Glucosylceramid... |
