Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase a tissue uptake and improves substrate reduction in fabry mice

Molecular Therapy

Volumn 20, Issue 4, 2012, Pages 717-726

  Benjamin, Elfrida R ^a   Khanna, Richie ^a   Schilling, Adriane ^a   Flanagan, John J ^a   Pellegrino, Lee J ^a   Brignol, Nastry ^a   Lun, Yi ^a   Guillen, Darlene ^a   Ranes, Brian E ^a   Frascella, Michelle ^a   Soska, Rebecca ^a   Feng, Jessie ^a   Dungan, Leo ^a   Young, Brandy ^b   Lockhart, David J ^a   Valenzano, Kenneth J ^a  

^a Amicus Therapeutics Inc   (United States)

^b UNIVERSITY OF CAPE TOWN   (South Africa)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GALACTOSIDASE; GR 181413A; LARAZOTIDE; MIGALASTAT; RECOMBINANT ALPHA GALACTOSIDASE; RECOMBINANT ENZYME; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL ISOLATION; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG BINDING; DRUG BLOOD LEVEL; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG HALF LIFE; DRUG STABILITY; DRUG TISSUE LEVEL; DRUG UPTAKE; ENZYME ACTIVITY; ENZYME INACTIVATION; ENZYME REPLACEMENT; ENZYME SUBSTRATE; FABRY DISEASE; FIBROBLAST; HUMAN; HUMAN CELL; IN VITRO STUDY; KNOCKOUT MOUSE; MALE; MONOTHERAPY; MOUSE; NONHUMAN; PROTEIN DENATURATION; RAT;

MUS; RATTUS;

EID: 84859439223     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.271     Document Type: Article

References (41)

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