A Phase 2 study of migalastat hydrochloride in females with Fabry disease: Selection of population, safety and pharmacodynamic effects

Molecular Genetics and Metabolism

Volumn 109, Issue 1, 2013, Pages 86-92

  Giugliani, R ^a   Waldek, S ^b   Germain, D P ^c   Nicholls, K ^d   Bichet, D G ^e   Simosky, J K ^f   Bragat, A C ^f   Castelli, J P ^f   Benjamin, E R ^f   Boudes, P F ^f  

^a HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^b SALFORD ROYAL NHS FOUNDATION TRUST   (United Kingdom)

^c LABORATOIRE DES SCIENCES DU CLIMAT ET DE L ENVIRONNEMENT   (France)

^d ROYAL MELBOURNE HOSPITAL   (Australia)

^e HÔPITAL DU SACRÉ COEUR DE MONTRÉAL   (Canada)

^f Amicus Therapeutics   (United States)

Author keywords

Galactosidase A; Fabry disease; Globotriaosylceramide; Lysosomal storage disorders; Pharmacological chaperone

Indexed keywords

ALPHA GALACTOSIDASE; GLOBOTRIAOSYLCERAMIDE; MIGALASTAT;

ABDOMINAL PAIN; ADULT; AGED; ARTHRALGIA; ARTICLE; BLOOD CELL; CLINICAL ARTICLE; CONTROLLED STUDY; DRUG EFFECT; DRUG SAFETY; DRUG TOLERABILITY; ELECTROCARDIOGRAM; ENZYME ACTIVITY; FABRY DISEASE; FEMALE; GENE MUTATION; GLOMERULUS FILTRATION RATE; HEADACHE; HEART LEFT VENTRICLE MASS; HISTOPATHOLOGY; HUMAN; HUMAN CELL; HUMAN TISSUE; KIDNEY; KIDNEY TUBULE; PAIN; PATIENT SELECTION; PERIPHERAL EDEMA; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; RHINOPHARYNGITIS; SIDE EFFECT; SKIN; URINALYSIS; VOMITING;

1-DEOXYNOJIRIMYCIN; ADULT; ALPHA-GALACTOSIDASE; ENZYME INHIBITORS; EPITHELIAL CELLS; FABRY DISEASE; FEMALE; HEK293 CELLS; HUMANS; KIDNEY; MIDDLE AGED; MUTATION; SKIN; TRANSFECTION;

EID: 84876084462     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.01.009     Document Type: Article

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