Current status of drug therapy development for Niemann-Pick type C disease

Drugs of the Future

Volumn 34, Issue 4, 2009, Pages 315-331

  Helquist, P ^a   Wiest, O ^a  

^a UNIVERSITY OF NOTRE DAME   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

2 HYDROXYPROPYL BETA CYCLODEXTRIN; 3ALPHA HYDROXY 5ALPHA PREGNAN 20 ONE; 3BETA (2 DIETHYLAMINOETHOXY)ANDROST 5 EN 17 ONE; AD 2765; CHOLESTEROL; CLOFIBRATE; COLESTYRAMINE; CP 340868; EZETIMIBE; HYPOCHOLESTEROLEMIC AGENT; IMATINIB; LIPID; MEVINOLIN; MIGLUSTAT; N (2,2,2 TRIFLUOROETHYL) N [4 [2,2,2 TRIFLUORO 1 HYDROXY 1 (TRIFLUOROMETHOXY)ETHYL]PHENYL]BENZENESULFONAMIDE; N(G) NITROARGININE METHYL ESTER; NICOTINIC ACID; NIFEDIPINE; OXYSTEROL; PERFLUOROOCTANOIC ACID; PHTHALIC ACID BIS(2 ETHYLHEXYL) ESTER; PRASTERONE; PRAVASTATIN; PROBUCOL; SPHINGOLIPID; T 1317; TOLNAFTATE; TRICYCLO[4.3.0.1]DECAN 9 YL XANTHOGENATE; UNCLASSIFIED DRUG; UNINDEXED DRUG; VALPROIC ACID; VORINOSTAT;

ABDOMINAL DISTENSION; ABDOMINAL PAIN; CUTANEOUS T CELL LYMPHOMA; DECREASED APPETITE; DIARRHEA; DRUG EFFICACY; ENDOSOME; FATALITY; FLATULENCE; GENE; GENE MUTATION; HUMAN; LIPID TRANSPORT; LIVER DYSFUNCTION; LYSOSOME; NAUSEA; NERVE DEGENERATION; NEUROPROTECTION; NIEMANN PICK DISEASE; NONHUMAN; NPC1 GENE; NPC2 GENE; PHENOTYPE; RARE DISEASE; REVIEW; SIDE EFFECT; SINGLE DRUG DOSE; SPLEEN DISEASE; T CELL LYMPHOMA; TREMOR; WEIGHT REDUCTION;

EID: 76649092600     PISSN: 03778282     EISSN: None     Source Type: Journal    
DOI: 10.1358/dof.2009.034.04.1352412     Document Type: Review

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