Changes in plasma and urine globotriaosylceramide levels do not predict Fabry disease progression over 1 year of agalsidase alfa

Genetics in Medicine

Volumn 15, Issue 12, 2013, Pages 983-989

  Schiffmann, Raphael ^a   Ries, Markus ^b   Blankenship, Derek ^c   Nicholls, Kathy ^d   Mehta, Atul ^e   Clarke, Joe T R ^f   Steiner, Robert D ^g   Beck, Michael ^h   Barshop, Bruce A ⁱ   Rhead, William ^j   West, Michael ^k   Martin, Rick ^l   Amato, David ^m   Nair, Nitin ^l   Huertas, Pedro ^n,o  

^a BAYLOR UNIVERSITY MEDICAL CENTER   (United States)

^b UNIVERSITY HOSPITAL HEIDELBERG   (Germany)

^c BAYLOR HEALTH CARE SYSTEM   (United States)

^d ROYAL MELBOURNE HOSPITAL   (Australia)

^e ROYAL FREE HOSPITAL   (United Kingdom)

^f HOSPITAL FOR SICK CHILDREN   (Canada)

^g OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^h JOHANNES GUTENBERG UNIVERSITY   (Germany)

ⁱ UNIVERSITY OF CALIFORNIA   (United States)

^j MEDICAL COLLEGE OF WISCONSIN   (United States)

^k DALHOUSIE UNIVERSITY   (Canada)

^l Shire Human Genetic Therapies   (United States)

^m VERTEX PHARMACEUTICALS   (United States)

ⁿ PFIZER INC   (United States)

^o HARVARD MIT DIVISION OF HEALTH SCIENCES AND TECHNOLOGY   (United States)

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Author keywords

agalsidase alfa; biomarkers; enzyme replacement therapy; Fabry disease; globotriaosylceramide

Indexed keywords

AGALSIDASE ALFA; GLOBOTRIAOSYLCERAMIDE; NONOXINOL 9;

ARTICLE; DIASTOLIC BLOOD PRESSURE; DISEASE COURSE; ENZYME REPLACEMENT; FABRY DISEASE; GLOMERULUS FILTRATION RATE; HEART LEFT VENTRICLE HYPERTROPHY; HEART LEFT VENTRICLE MASS; HUMAN; KIDNEY FUNCTION; LIPID BLOOD LEVEL; META ANALYSIS; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PREDICTIVE VALUE; PROTEIN URINE LEVEL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SYSTOLIC BLOOD PRESSURE; URINALYSIS; URINARY EXCRETION;

ADULT; ALPHA-GALACTOSIDASE; BIOLOGICAL MARKERS; DISEASE PROGRESSION; DOUBLE-BLIND METHOD; FABRY DISEASE; GLOMERULAR FILTRATION RATE; HEART VENTRICLES; HUMANS; ISOENZYMES; MALE; MIDDLE AGED; RENAL INSUFFICIENCY, CHRONIC; TREATMENT OUTCOME; TRIHEXOSYLCERAMIDES; YOUNG ADULT;

EID: 84890084688     PISSN: 10983600     EISSN: 15300366     Source Type: Journal    
DOI: 10.1038/gim.2013.56     Document Type: Article

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