Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders

Assay and Drug Development Technologies

Volumn 9, Issue 3, 2011, Pages 213-235

  Valenzano, Kenneth J ^a   Khanna, Richie ^a   Powe, Allan C ^b   Boyd, Robert ^a   Lee, Gary ^a   Flanagan, John J ^a   Benjamin, Elfrida R ^a  

^a Amicus Therapeutics   (United States)

^b ArunA Biomedical Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

1 DEOXYNOJIRIMYCIN; 2 DECANOYLAMINO 3 MORPHOLINO 1 PHENYL 1 PROPANOL; 5 [(4 METHYLPHENYL)THIO]QUINAZOLINE 2,4 DIAMINE; ADAMANTANE DERIVATIVE; ADAMANTYL AMIDE; AFEGOSTAT; AGALSIDASE ALFA; AGALSIDASE BETA; AMBROXOL; CASTANOSPERMINE; DILTIAZEM; G PROTEIN COUPLED RECEPTOR; GALACTOSAMINE; GALSULFASE; HEXANOIC ACID; IDURONATE 2 SULFATASE; IMIGLUCERASE; ION CHANNEL; LARAZOTIDE; LARONIDASE; LUMIZYME; LYSOSOME ENZYME; MIGALASTAT; MIGLUSTAT; N BUTYL 1 DEOXYNOJIRIMYCIN; N BUTYL 1-DEOXYGALACTONOJIRIMYCIN; PIPERIDINE DERIVATIVE; QUINAZOLINE DERIVATIVE; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; TRANSCRIPTION FACTOR; UNCLASSIFIED DRUG; UNINDEXED DRUG; VELAGLUCERASE ALFA;

CELL ACTIVITY; CELL MIGRATION; DOSE RESPONSE; DRUG MECHANISM; ENDOPLASMIC RETICULUM; ENZYME ACTIVITY; ENZYME DEFICIENCY; ENZYME INHIBITION; ENZYME REPLACEMENT; FABRY DISEASE; GAUCHER DISEASE; GENE MUTATION; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; LYSOSOME STORAGE DISEASE; MAROTEAUX LAMY SYNDROME; NONHUMAN; PHARMACOGENETICS; REVIEW;

ANIMALS; BIOLOGICAL ASSAY; DRUG DESIGN; HUMANS; LYSOSOMAL STORAGE DISEASES; MOLECULAR CHAPERONES; TECHNOLOGY, PHARMACEUTICAL;

EID: 79957628617     PISSN: 1540658X     EISSN: 15578127     Source Type: Journal    
DOI: 10.1089/adt.2011.0370     Document Type: Review

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