Substrate reduction therapy for lysosomal storage diseases

Acta Paediatrica, International Journal of Paediatrics, Supplement

Volumn 94, Issue 447, 2005, Pages 69-75

  Cox, T M ^a,b  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^b ADDENBROOKE S HOSPITAL   (United Kingdom)

Author keywords

Gaucher disease; Lysosomal storage diseases; Miglustat; N butyldeoxynojirimycin; Substrate reduction therapy

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE A; CERAMIDE GLUCOSYLTRANSFERASE INHIBITOR; GANGLIOSIDE GM2; GLUCOSYLCERAMIDE; GLUCOSYLTRANSFERASE; GLYCOSPHINGOLIPID; IMIGLUCERASE; MIGLUSTAT; MORPHOLINE DERIVATIVE; N BUTYLDEOXYGALACTONOJIRIMYCIN; TRANSFERASE INHIBITOR; UNCLASSIFIED DRUG; URIDINE DIPHOSPHATE; CARBOHYDRATE DERIVATIVE; CERAMIDE GLUCOSYLTRANSFERASE; CHOLESTEROL; IMINOSUGAR; LACTOSYLCERAMIDE; LIPOPROTEIN; SPHINGOLIPID;

BLOATING; CELL MEMBRANE; CELL ORGANELLE; CLINICAL TRIAL; CONFERENCE PAPER; DIARRHEA; DRUG APPROVAL; DRUG BLOOD LEVEL; DRUG EFFECT; DRUG EFFICACY; DRUG EXCRETION; DRUG MARKETING; DRUG MECHANISM; DRUG MEGADOSE; DRUG PENETRATION; DRUG TOLERABILITY; ENDOPLASMIC RETICULUM; ENZYME INHIBITION; ENZYME REPLACEMENT; ENZYME SUBSTRATE; FABRY DISEASE; FLATULENCE; FOOD AND DRUG ADMINISTRATION; GAUCHER DISEASE; GM1 GANGLIOSIDOSIS; GM2 GANGLIOSIDOSIS; HUMAN; LIPOGENESIS; LYSOSOME STORAGE DISEASE; NEUROPATHY; NIEMANN PICK DISEASE; NONHUMAN; PARESTHESIA; PRIORITY JOURNAL; SANDHOFF DISEASE; SANFILIPPO SYNDROME; TREMOR; ATHEROSCLEROSIS; CHOLESTEROL SYNTHESIS; DEGENERATIVE DISEASE; DRUG INDICATION; LIPOPROTEIN METABOLISM; METABOLIC DISORDER; NEUROPATHIC PAIN; PATIENT SELECTION; SUBSTRATE REDUCTION THERAPY;

1-DEOXYNOJIRIMYCIN; ENZYME INHIBITORS; GLUCOSYLCERAMIDASE; GLUCOSYLCERAMIDES; GLYCOSIDE HYDROLASES; GLYCOSPHINGOLIPIDS; HUMANS; LYSOSOMAL STORAGE DISEASES; SUBSTRATE SPECIFICITY;

EID: 16844368149     PISSN: 08035326     EISSN: None     Source Type: Journal    
DOI: 10.1080/08035320510028157     Document Type: Conference Paper

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