Substrate-reduction therapy with miglustat for glycosphingolipid storage disorders affecting the brain

Expert Review of Endocrinology and Metabolism

Volumn 4, Issue 3, 2009, Pages 217-224

  Lachmann, Robin H ^a  

^a NATIONAL HOSPITAL FOR NEUROLOGY AND NEUROSURGERY   (United Kingdom)

Author keywords

Enzymes replacement therapy; Gaucher disease; Glycosphingolipidosis; GM2 gangliosidosis; Lysosomal storage disorder; Miglustat; Niemann pick disease; Sandhoff disease; Substrate reduction therapy; Tay Sachs disease

Indexed keywords

2 DECANOYLAMINO 3 MORPHOLINO 1 PHENYL 1 PROPANOL; BETA ADRENERGIC RECEPTOR BLOCKING AGENT; GANGLIOSIDE GM1; GANGLIOSIDE GM2; GLUCOSYLCERAMIDE; GLYCOSPHINGOLIPID; IMIGLUCERASE; MIGLUSTAT; PLACEBO;

BLOOD BRAIN BARRIER; CLINICAL TRIAL; DIARRHEA; DISEASE CLASSIFICATION; DOSE RESPONSE; DRUG ABSORPTION; DRUG CLEARANCE; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG HALF LIFE; DRUG MEGADOSE; DRUG METABOLISM; DRUG PENETRATION; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; ENZYME ACTIVITY; ENZYME REPLACEMENT; FABRY DISEASE; GASTROINTESTINAL SYMPTOM; GAUCHER DISEASE; GAUCHER DISEASE TYPE 1; GM1 GANGLIOSIDOSIS; GM2 GANGLIOSIDOSIS; HUMAN; LIPID DEGRADATION; LIPID STORAGE; LIPIDOSIS; LIPOGENESIS; LYSOSOME STORAGE DISEASE; MAXIMUM PLASMA CONCENTRATION; MUCOPOLYSACCHARIDOSIS; NIEMANN PICK DISEASE; NONHUMAN; PERIPHERAL NEUROPATHY; PRIORITY JOURNAL; REVIEW; SACCADIC EYE MOVEMENT; SANDHOFF DISEASE; SUBSTRATE REDUCTION THERAPY; TAY SACHS DISEASE; TREMOR;

EID: 70049098302     PISSN: 17446651     EISSN: None     Source Type: Journal    
DOI: 10.1586/eem.09.8     Document Type: Review

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