gaucher

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.

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 mucoactive drug with several properties including secretolytic and secretomotoric actions that restore the physiological clearance mechanisms of the respiratory tract which play an important role in the body's natural defense mechanisms. It stimulates synthesis and release of surfactant by type II pneumocytes. Surfactants act as an anti-glue factor by reducing the adhesion of mucus to the bronchial wall, in improving its transport and in providing protection against infection and irritating agents.

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.

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.

Miglustat hydrochloride (N-Butyldeoxynojirimycin hydrochloride) is an inhibitor of glucosylceramide synthase and can be used for studies about Type I Gaucher disease.

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).

Glucocerebrosides (Gaucher cerebroside), a compound isolated from the fruit of Citrus unshiu, is a major component of the endomembrane system and plasma membrane of most eukaryotic cells and antagonizes insulin signaling.

Eliglustat (Genz 99067) is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease.

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 glucocerebrosidase activity in a pH-dependent manner. It activates glucocerebrosidase to decrease glucosylceramide accumulation in fibroblasts and lymphoblasts isolated from patients with Gaucher and Fabry disease, respectively.

Ibiglustat (Venglustat) succinate is an orally active, brain-penetrant inhibitor of glucosylceramide synthase (GCS) utilized in the investigation of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease.

Sphinganine 1-phosphate is an intermediate in the sphingolipid metabolic pathway and is structurally similar to sphingosine. It has a protective effect on the kidney and liver through activation of the S1P1 receptor in mice. D-erythro-Dihydrosphingosine 1-phosphate acts as an agonist for S1P4 in Homo sapiens and may activate GPR63 (G protein-coupled receptor), mainly used in the study of Fabry disease, Gaucher disease, Krabbe disease and Leukodystrophy.

Glucocerebrosidase-IN-1 (compound 11a) is a potent and selective GCase (glucocerebrosidase) inhibitor, with an IC50 of 29.3 μM and a Ki of 18.5 μM. Due to GCase involvement in pathological disorders consequent to enzyme deficiency, Glucocerebrosidase-IN-1 can be used for research of Gaucher disease (GD) and Parkinson's disease (PD)[1].

Glucosylceramide synthase-IN-1 (T-036) is a potent, orally active, blood-brain barrier permeable glucosylceramide synthase (GCS) inhibitor, acting on human GCS (IC50: 31 nM) and mouse GCS (IC50: 51 nM), and can be used to study Gaucher disease.

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][2].

Glucosylsphingosine (Lyso-Gb1) is a naturally occurring glycosyl ceramide, a metabolite of GBA (acid β-glucosidase), involved in cellular recognition, signaling, and intercellular interactions.Glucopsychosine accumulates in certain neurodegenerative diseases (e.g., Gaucher disease), disrupts lysosomal function through activation of mammalian target of rapamycin complex 1 (mTORC1), and is a noncompetitive inhibitor of glucocerebrosidase.

Glucocerebrosidase-IN-1 (compound 11a) hydrochloride is a potent and selective glucocerebrosidase (GCase) inhibitor with IC50 of 29.3 μM and Ki of 18.5 μM, used in the research of Gaucher disease (GD) and Parkinson's disease (PD) [1].

Therapeutic Agent-1, a heteroaryl compound, serves as an enzyme replacement therapy to enhance glucocerebrosidase activity in the treatment of Gaucher disease [1].

Ambroxol acefylline, an active metabolite of the proagent Bromhexine, has potent expectorant effects and functions as a glucocerebrosidase (GCase) chaperone, increasing GCase activity. It induces lung autophagy and has potential applications in Parkinson's disease and neuronopathic Gaucher disease research [1][2].

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 compound shows promise for Gaucher disease research.