The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases

Journal of Lipid Research

Volumn 55, Issue 7, 2014, Pages 1215-1225

  Shayman, James A ^a   Larsen, Scott D ^b  

^a Chemistry   (United States)

^b UNIVERSITY OF MICHIGAN   (United States)

Author keywords

Ambroxol; Eliglustat tartrate; Glucosylceramide; Isofagomine; Lysosome; Miglustat; Pharmacological chaperone; Pyrimethamine

Indexed keywords

2 (2,3 DIHYDRO 1H INDEN 2 YL) N [1 (2,3 DIHYDROBENZO[B][1,4]DIOXIN 6 YL) 1 HYDROXY 3 (PYRROLIDIN 1 YL)PROPAN 2 YL]ACETAMIDE; 2 DECANOYLAMINO 3 MORPHOLINO 1 PHENYL 1 PROPANOL; ACETAMIDE DERIVATIVE; ADAMANTANE DERIVATIVE; AFEGOSTAT; AGENTS AFFECTING LIPID METABOLISM; AMBROXOL; DEXTRO THREO 1 ETHYLENEDIOXYPHENYL 2 PALMITOYL 3 PYRROLIDINO PROPANOL; ELIGLUSTAT; ENZYME INHIBITOR; EXEL 0346; GLYCOSPHINGOLIPID; GZ 161; IMIGLUCERASE; IMINOSUGAR; MIGALASTAT; MIGLUSTAT; N (5 ADAMANTANE 1 YL METHOXYPENTYL)DEOXYNOJIRIMYCIN; PLACEBO; PYRIMETHAMINE; UNCLASSIFIED DRUG; CHAPERONE; GLYCOSIDASE; GLYCOSIDASE INHIBITOR;

CLINICAL FEATURE; DISEASE MODEL; DRUG APPROVAL; DRUG BIOAVAILABILITY; DRUG BLOOD LEVEL; DRUG DEVELOPMENT; DRUG DISTRIBUTION; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG POTENCY; DRUG RESPONSE; DRUG SAFETY; DRUG SCREENING; DRUG TISSUE LEVEL; DRUG TOLERABILITY; DRUG URINE LEVEL; DRUG USE; ENZYME INHIBITION; ENZYME REPLACEMENT; EUROPEAN MEDICINES AGENCY; FABRY DISEASE; FOOD AND DRUG ADMINISTRATION; GAUCHER DISEASE; GM1 GANGLIOSIDOSIS; GM2 GANGLIOSIDOSIS; HUMAN; IC 50; LIPID METABOLISM; LIPOGENESIS; LIPOLYSIS; LYSOSOME STORAGE DISEASE; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; SANDHOFF DISEASE; TAY SACHS DISEASE; ANIMAL; GENETICS; METABOLISM; MUTATION; PATHOLOGY;

ANIMALS; GLYCOSIDE HYDROLASE INHIBITORS; GLYCOSIDE HYDROLASES; GLYCOSPHINGOLIPIDS; HUMANS; LYSOSOMAL STORAGE DISEASES; MOLECULAR CHAPERONES; MUTATION;

EID: 84903985741     PISSN: 00222275     EISSN: 15397262     Source Type: Journal    
DOI: 10.1194/jlr.R047167     Document Type: Review

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