A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: Safety and tolerability

Genetics in Medicine

Volumn 16, Issue 5, 2014, Pages 359-366

  Pastores, Gregory M ^a   Rosenbloom, Barry ^b   Weinreb, Neal ^c   Goker Alpan, Ozlem ^d   Grabowski, Gregory ^e   Cohn, Gabriel M ^f   Zahrieh, David ^f  

^a NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b Cedars Sinai Tower Hematology Oncology   (United States)

^c University Research Foundation for Lysosomal Storage Diseases   (United States)

^d Lysosomal Research and Treatment Unit   (United States)

^e CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^f SHIRE   (United States)

Author keywords

antidrug antibodies; enzyme replacement therapy; imiglucerase; type 1 Gaucher disease; velaglucerase alfa

Indexed keywords

IMIGLUCERASE; MONOCLONAL ANTIBODY; NEUTRALIZING ANTIBODY; VELAGLUCERASE ALFA; ANTIBODY; GLUCOSYLCERAMIDASE; RECOMBINANT PROTEIN; VELAGLUCERASE ALFA, HUMAN;

ADOLESCENT; ADULT; AGED; ANTIBODY DETECTION; ARTICLE; AUTOSOMAL RECESSIVE INHERITANCE; BACKACHE; CHILD; CLINICAL PROTOCOL; DRUG INFUSION; DRUG SAFETY; DRUG SUBSTITUTION; DRUG TOLERABILITY; DRUG WITHDRAWAL; ENZYME REPLACEMENT; FATIGUE; FEMALE; GAUCHER DISEASE; HEADACHE; HUMAN; INFUSION RELATED REACTION; LYSOSOME STORAGE DISEASE; MAJOR CLINICAL STUDY; MALE; MIGRAINE; MULTICENTER STUDY; NAUSEA; OPEN STUDY; PHASE 3 CLINICAL TRIAL (TOPIC); RADIOIMMUNOPRECIPITATION; RHINOPHARYNGITIS; BLOOD; CLINICAL TRIAL; IMMUNOLOGY; MIDDLE AGED; VERY ELDERLY; YOUNG ADULT;

ADOLESCENT; ADULT; AGED; AGED, 80 AND OVER; ANTIBODIES; CHILD; ENZYME REPLACEMENT THERAPY; FEMALE; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; HUMANS; MALE; MIDDLE AGED; RECOMBINANT PROTEINS; YOUNG ADULT;

EID: 84899982247     PISSN: 10983600     EISSN: 15300366     Source Type: Journal    
DOI: 10.1038/gim.2013.154     Document Type: Article

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