Substrate reduction therapy for glycosphingolipid storage disorders

Expert Opinion on Investigational Drugs

Volumn 10, Issue 3, 2001, Pages 455-466

  Lachmann, R H ^a   Platt, F M ^a  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

Author keywords

Gaucher's disease; Glycosphingolipid lysosomal storage diseases; Glycosphingolipids; Imino sugar; N butyldeoxynojirimycin; Substrate deprivation; Substrate reduction

Indexed keywords

ALGLUCERASE; ALPHA GALACTOSIDASE; BETA N ACETYLHEXOSAMINIDASE A; CERAMIDE; CERAMIDE GLUCOSYLTRANSFERASE; GALACTOSYLCERAMIDE; GANGLIOSIDE GM1; GANGLIOSIDE GM2; GLOBOTRIAOSYLCERAMIDE; GLUCOSE; GLUCOSYLCERAMIDASE; GLUCOSYLTRANSFERASE; GLYCOLIPID; GLYCOSPHINGOLIPID; LACTOSYLCERAMIDE; MIGLUSTAT; PALMITOYL COENZYME A; SPHINGANINE; SPHINGOMYELIN; URIDINE DIPHOSPHATE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; BIOSYNTHESIS; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DIARRHEA; DRUG BLOOD LEVEL; DRUG EFFICACY; DRUG MECHANISM; DRUG SAFETY; DRUG STRUCTURE; ENZYME INHIBITION; FABRY DISEASE; FLATULENCE; GASTROINTESTINAL TRACT; GAUCHER DISEASE; HEPATOSPLENOMEGALY; HUMAN; IN VITRO STUDY; KNOCKOUT MOUSE; MOUSE; NAUSEA; NONHUMAN; PARESTHESIA; REVIEW; SANDHOFF DISEASE; TARGET ORGAN; TAY SACHS DISEASE; WEIGHT REDUCTION;

1-DEOXYNOJIRIMYCIN; ANIMALS; GLYCOSPHINGOLIPIDS; HUMANS; LYSOSOMAL STORAGE DISEASES; MICE;

EID: 0035110728     PISSN: 13543784     EISSN: None     Source Type: Journal    
DOI: 10.1517/13543784.10.3.455     Document Type: Review

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