Pharmacological chaperones as therapeutics for lysosomal storage diseases

Journal of Medicinal Chemistry

Volumn 56, Issue 7, 2013, Pages 2705-2725

  Boyd, Robert E ^a   Lee, Gary ^a   Rybczynski, Philip ^a   Benjamin, Elfrida R ^a   Khanna, Richie ^a   Wustman, Brandon A ^b   Valenzano, Kenneth J ^a  

^a Amicus Therapeutics   (United States)

^b Amicus Therapeutics   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

1 ADAMANTYLCARBOXAMIDE; 1 DEOXYNOJIRIMYCIN; 2,5 DIDEOXY 2,5 IMINO D ALTRITOL; ACETAMIDODIDEOXYNOJIRIMYCIN; AGALSIDASE ALFA; AGALSIDASE BETA; ALGLUCERASE; ALPHA GALACTOSIDASE; ALPHA GLUCOSIDASE; AMBROXOL; AT 2220; AZASUGAR; BETA N ACETYLHEXOSAMINIDASE; CARBOHYDRATE DERIVATIVE; CASTANOSPERMINE; CASTANOSPERMINE DERIVATIVE; CHAPERONE; GALACTOSE; GALSULFASE; GR 181413A; GUANIDINE DERIVATIVE; IDURONATE 2 SULFATASE; IMIGLUCERASE; IMINOSUGAR; ISOFAGAMINE; LARAZOTIDE; LARONIDASE; LYSOSOME ENZYME; MIGALASTAT; N (N NONYL)DEOXYNOJIRIMYCIN; N ACETYLGLUCOSAMINE THIAZOLINE; N BUTYL DEOXYNOJIRIMYCIN; N OCTYLVALIENAMINE; PYRIMETHAMINE; PYRROLIDINE DERIVATIVE; QUINAZOLINE DERIVATIVE; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; TALIGLUCERASE ALFA; UNCLASSIFIED DRUG; UNINDEXED DRUG; VELAGLUCERASE ALFA;

AMENORRHEA; ATAXIA; BLURRED VISION; CONCENTRATION RESPONSE; CONSCIOUSNESS DISORDER; DEGENERATIVE DISEASE; DRUG DOSE ESCALATION; DRUG EFFECT; DRUG PROTEIN BINDING; DRUG STRUCTURE; ENZYME ACTIVITY; ENZYME STABILITY; FABRY DISEASE; GAUCHER DISEASE; GENE MUTATION; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; LYSOSOME STORAGE DISEASE; MUCOPOLYSACCHARIDOSIS TYPE C; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); REVIEW; SANDHOFF DISEASE; SANFILLIPO SYNDROME; SANTAVUORI HALTIA DISEASE; STRUCTURE ACTIVITY RELATION; TAY SACHS DISEASE; TREMOR; UNSPECIFIED SIDE EFFECT; WEAKNESS;

CELL LINE; GLYCOLIPIDS; HUMANS; LYSOSOMAL STORAGE DISEASES; MOLECULAR CHAPERONES; STRUCTURE-ACTIVITY RELATIONSHIP;

EID: 84876225140     PISSN: 00222623     EISSN: 15204804     Source Type: Journal    
DOI: 10.1021/jm301557k     Document Type: Review

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