WUSTMAN, Brandon,VALENZANO, Kenneth,BOYD, Robert,WUSTMAN BRANDON,WUSTMAN, BRANDON,VALENZANO KENNETH,VALENZANO, KENNETH,BOYD ROBERT,BOYD, ROBERT
申请号：
ES15173518
公开号：
ES2646099T3
申请日:
2010.10.12
申请国别(地区):
ES
年份:
2017
代理人:
摘要：
A pharmacological chaperone for use in treating Cerebral Amyloid Angiopathy (CAA) where the pharmacological chaperone selectively binds to and stabilizes a gangliosidase selected from the group consisting of ß-hexosaminidase A, ß-hexosaminidase B and ß-hexosaminidase S； and wherein the pharmacological chaperone is selected from: N-butyl-deoxygalactonojirimycin (NB-DGJ) N-acetyl-glucosamine-thiazoline (NGT) 2-acetamido-1,2-dideoxyinojirimycin (AdDNJ) 2-acetamido-2-deoxinojirimycin ( ADNJ) 6-acetamido-6-deoxystanspermine Pyrimethamine 2-acetamido-1,4-imino-1,2,4-trideoxy-L-arabinitol (LABNAc) N-benzyl 2-acetamido-1,4-imino-1,2 , 4-trideoxy-L-arabinitol (NBn-LABNAc) N-butyl 2-acetamido-1,4-imino-1,2,4-trideoxy-L-arabinitol (NBu-LABNAc) DABNAc hydrochloride (2R, 3R, 4S, 5R) -2-Acetamido-3,4-dihydroxy-5-hydroxy-methyl-piperidinium (GalNAc-isophagomine HCl) Galactose O- (2-acetamido-2-deoxy-d-glucopyranosylidene) -amino-N-phenylcarbamate (Gal-PUGNAc) NAG-thiazoline (and its derivatives) PUGNAc (and its derivatives) 6-acetamido-2,6-dideoxy-2-C-hydroxymethyl-D-gluco-δ-lactam 2-acetamido-2-deoxy- d-glucono-d-lactam 2-acetamido-2-deoxy-d-glucono-deocinojirimycin derived from 3-hydroxypipecolic acid derived from 3,4,5-trihydroxypiolic acid (3R, 4R, 5R, 6R) -tetrahydroxyazepane NAc-1-Cp NAc-1- (CH3) 2 (5R, 6S, 7S, 8S) -8-acetamido-5,6,7,8-tetrahydro-5- (hydroxymethyl) -imidazole [1,2 -a] pyridine-6,7-triol 2-acetamido-1,2,5-trideoxy-1,5-imino-D-glucitol (2R, 3R, 4S, 5R) -2-acetamido-5-aminomethyl-3 , 4-dihydroxy-piperidine (2R, 3R, 4S, 5R) -2-acetamido-5-hydroxymethyl-3,4-dihydroxy-piperidine.Una chaperona farmacológica para uso en tratar la Angiopatía Amiloide Cerebral (CAA) en donde la chaperona farmacológica se une selectivamente a y estabiliza una gangliosidasa seleccionada del grupo que consiste en ß- hexosaminidasa A, ß-hexosaminidasa B y ß-hexosaminidasa S； y en donde la chaperona farmacológica se selecciona de: N-butil-desoxigalactonojirimicina (NB-DGJ) N-acetil-glucosa
