gaucher — TargetMol Chemicals

Cat No.	Product Name	Synonyms	Targets
T8986	ML198		Glucokinase
	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.		Glucokinase
T6234	Ambroxol hydrochloride	Mucosolvan,Mucoangin,Ambroxol HCl	Sodium Channel , Autophagy
	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.		Sodium Channel , Autophagy
T0920	Ambroxol	Ambroxolum,NA-872	P450 , Autophagy
	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...		P450 , Autophagy
T12039	Miglustat	N-Butyldeoxynojirimycin,OGT918,NB-DNJ	Others
	Miglustat (NB-DNJ) is an alkylated product of imino sugar deoxynojirimycin.It is an inhibitor of glucosylceramide synthase, primarily to treat Type I Gaucher disease (GD1).		Others
TQ0155	Miglustat hydrochloride	NB-DNJ hydrochloride,N-Butyldeoxynojirimycin hydrochloride,OGT918 hydrochloride	Transferase
	Miglustat hydrochloride (N-Butyldeoxynojirimycin hydrochloride) is an inhibitor of glucosylceramide synthase and can be used for studies about Type I Gaucher disease.		Transferase
T36582	Glucocerebrosides (Gaucher's spleen)
	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...
T3663	Eliglustat	GENZ-112638,Genz 99067	Transferase
	Eliglustat (Genz 99067) is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease.		Transferase
T40544	NCGC00092410	ML008	Glucosidase
	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.		Glucosidase
T8990	ML266		Glucokinase
	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...		Glucokinase
T37440	C2 Adamantanyl Galactosylceramide (d18:1/2:0)
	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...
T73716	Miglustat-d
	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]...
T78493	Glucosylsphingosine	Lyso-Gb1,Glucopsychosine
	Glucosylsphingosine (lyso-Gb1), a deacylated glucosylceramide derivative metabolized by glucocerebrosidase, serves as a promising, reliable, and specific biomarker for Gaucher disease monitoring [1].
T39105	Ibiglustat succinate	GZ402671succinate,Venglustat succinate,Ibiglustat succinate,SAR402671succinate
	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 ...
T80994	Therapeutic agent-1
	Therapeutic Agent-1, a heteroaryl compound, serves as an enzyme replacement therapy to enhance glucocerebrosidase activity in the treatment of Gaucher disease [1].
T78671	Glucocerebrosidase-IN-1 hydrochloride		Glucosidase
	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...		Glucosidase
T60353	Glucocerebrosidase-IN-1
	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...
T62898	Glucosylceramide synthase-IN-1
	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...