Substrate reduction therapy of glycosphingolipid storage disorders

Journal of Inherited Metabolic Disease

Volumn 29, Issue 2-3, 2006, Pages 449-456

  Aerts, Johannes M F G ^a   Hollak, Carla E M ^a   Boot, Rolf G ^a   Groener, Johanna E M ^a   Maas, Mario ^a  

^a UNIVERSITY OF AMSTERDAM   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

2 DECANOYLAMINO 3 MORPHOLINO 1 PHENYL 1 PROPANOL; ADAMANTANE PENTYLDEOXYNORIJIMYCIN; ALGLUCERASE; CERAMIDE GLUCOSYLTRANSFERASE INHIBITOR; GLUCOSYLCERAMIDE; GLYCOSPHINGOLIPID; IMIGLUCERASE; MIGLUSTAT; N BUTYLDEOXYGALACTONOJIRIMYCIN; RECOMBINANT ENZYME; RECOMBINANT GLUCOCEREBROSIDASE; UNCLASSIFIED DRUG;

ABSENCE OF SIDE EFFECTS; ANTIINFLAMMATORY ACTIVITY; ARTICLE; BLOATING; CLINICAL FEATURE; CLINICAL TRIAL; DIARRHEA; DISEASE SEVERITY; DRUG ACCUMULATION; DRUG PENETRATION; ENZYME REPLACEMENT; ENZYME SUBSTRATE; ENZYME SYNTHESIS; FABRY DISEASE; FLATULENCE; GASTROINTESTINAL TOXICITY; GAUCHER DISEASE; GENE REPLACEMENT THERAPY; GENE THERAPY; HUMAN; LONG TERM CARE; MACROPHAGE; MAINTENANCE THERAPY; NIEMANN PICK DISEASE; NONHUMAN; PATIENT MONITORING; PERIPHERAL NEUROPATHY; SANDHOFF DISEASE; STORAGE DISEASE; TAY SACHS DISEASE; TREATMENT OUTCOME; TREMOR;

1-DEOXYNOJIRIMYCIN; ANTI-INFLAMMATORY AGENTS; BIOLOGICAL MARKERS; CLINICAL TRIALS; CLINICAL TRIALS AS TOPIC; ENZYME INHIBITORS; GAUCHER DISEASE; GENE THERAPY; GLUCOSYLCERAMIDASE; GLUCOSYLTRANSFERASES; GLYCOSPHINGOLIPIDS; HUMANS; LYSOSOMAL STORAGE DISEASES; MORPHOLINES; PRACTICE GUIDELINES; PRACTICE GUIDELINES AS TOPIC; RECOMBINANT PROTEINS; TREATMENT OUTCOME;

EID: 33745112205     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-006-0272-5     Document Type: Article

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