Coformulation of a novel human α-galactosidase a with the pharmacological chaperone at1001 leads to improved substrate reduction in fabry mice

Molecular Therapy

Volumn 23, Issue 7, 2015, Pages 1169-1181

  Xu, Su ^a   Lun, Yi ^a   Brignol, Nastry ^a   Hamler, Rick ^a   Schilling, Adriane ^a   Frascella, Michelle ^a   Sullivan, Sean ^a   Boyd, Robert E ^a   Chang, Kate ^a   Soska, Rebecca ^a   Garcia, Anadina ^a   Feng, Jessie ^a   Yasukawa, Hidehito ^b   Shardlow, Carole ^c   Churchill, Alison ^c   Ketkar, Amol ^d   Robertson, Nicola ^c   Miyamoto, Masahito ^b   Mihara, Kazutoshi ^b   Benjamin, Elfrida R ^a   Lockhart, David J ^e   Hirato, Tohru ^b   Fowles, Susie ^c   Valenzano, Kenneth J ^a   Khanna, Richie ^a   more..

^a Amicus Therapeutics Inc   (United States)

^b JCR Pharmaceuticals Co Ltd   (Japan)

^c GLAXOSMITHKLINE   (United Kingdom)

^d GLAXOSMITHKLINE   (United States)

^e TranscripTx Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GALACTOSIDASE; GLOBOTRIAOSYLCERAMIDE; LARAZOTIDE; RECOMBINANT ALPHA GALACTOSIDASE; RECOMBINANT ENZYME; UNCLASSIFIED DRUG; AT-1001; CHAPERONE; DRUG COMBINATION; OLIGOPEPTIDE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG DISTRIBUTION; DRUG FORMULATION; DRUG HALF LIFE; DRUG POTENCY; DRUG STABILITY; ENDOTHELIUM CELL; ENZYME REPLACEMENT; FABRY DISEASE; FIBROBLAST; GLOMERULUS EPITHELIUM CELL; HEART MUSCLE CELL; HUMAN; HUMAN CELL; IN VITRO STUDY; KIDNEY TUBULE CELL; MALE; MAXIMUM PLASMA CONCENTRATION; MOUSE; NONHUMAN; PH; PROTEIN BLOOD LEVEL; VASCULAR ENDOTHELIUM; ANIMAL; DISEASE MODEL; DRUG COMBINATION; DRUG EFFECTS; ENZYME SPECIFICITY; MUTATION; PATHOLOGY;

MUS;

ALPHA-GALACTOSIDASE; ANIMALS; DISEASE MODELS, ANIMAL; DRUG COMBINATIONS; ENZYME REPLACEMENT THERAPY; FABRY DISEASE; FIBROBLASTS; HUMANS; MICE; MOLECULAR CHAPERONES; MUTATION; OLIGOPEPTIDES; SUBSTRATE SPECIFICITY;

EID: 84934435392     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.87     Document Type: Article

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