ML-198

ML-198 is a chemical chaperone of glucocerebrosidase. It activates hydrolysis of the fluorescent glucocerebrosidase substrate 4-methylumbelliferyl-beta-D-glucopyranoside (4-MUG, Cay-20948) in spleen homogenate isolated from patients with Gaucher disease with the homozygous N370S mutant of glucocerebrosidase (glucocerebrosidaseN370S, AC50 = 0.36 µM). ML-198 (5 µM) increases the translocation of glucocerebrosidaseN370S to lysosomes in dermal fibroblasts isolated from patients with Gaucher disease.Formal Name: N-(4-ethynylphenyl)-5,7-dimethyl-pyrazolo[1,5-a]pyrimidine-3-carboxamide. CAS Number: 1380716-06-2. Synonyms: NCGC758. Molecular Formula: C17H14N4O. Formula Weight: 290.3. Purity: >98%. Formulation: (Request formulation change), A solid. Solubility: DMSO: 5 mg/ml. lambdamax: 215, 244, 293 nm. SMILES: O=C(C1=C(N=C(C)C=C2C)N2N=C1)NC3=CC=C(C=C3)C#C. InChi Code: InChI=1S/C17H14N4O/c1-4-13-5-7-14(8-6-13)20-17(22)15-10-18-21-12(3)9-11(2)19-16(15)21/h1,5-10H,2-3H3,(H,20,22). InChi Key: HVZPJBKUFRREQC-UHFFFAOYSA-N.