Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase

Molecular Genetics and Metabolism

Volumn 117, Issue 2, 2016, Pages 164-171

  Smith, Laurie ^a   Rhead, William ^b   Charrow, Joel ^c   Shankar, Suma P ^d,l   Bavdekar, Ashish ^d   Longo, Nicola ^e   Mardach, Rebecca ^f   Harmatz, Paul ^g   Hangartner, Thomas ^h   Lee, Hak Myung ⁱ   Crombez, Eric ^j   Pastores, Gregory M ^k  

^a Center for Pediatric Genomic Medicine   (United States)

^b Children's Hospital of Wisconsin   (United States)

^c CHILDREN'S MEMORIAL HOSPITAL   (United States)

^d EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e KEM HOSPITAL   (India)

^f UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^g KAISER PERMANENTE   (United States)

^h CHILDREN S HOSPITAL OAKLAND   (United States)

ⁱ Wright State University   (United States)

^j Biostatistics and Statistical Programming Department   (United States)

^k Shire   (United States)

^l MATER MISERICORDIAE UNIVERSITY HOSPITAL   (Ireland)

more..

Author keywords

Children and adolescents; Enzyme replacement therapy; Gaucher disease; Pediatric patients; Velaglucerase alfa

Indexed keywords

IMIGLUCERASE; IMMUNOGLOBULIN G ANTIBODY; VELAGLUCERASE ALFA; GLUCOSYLCERAMIDASE;

ABSENCE OF SIDE EFFECTS; ADOLESCENT; ARTICLE; CHILD; CLINICAL ARTICLE; CONVULSION; DRUG EFFICACY; DRUG SAFETY; DRUG SUBSTITUTION; DRUG TOLERABILITY; ENZYME REPLACEMENT; EXPLORATORY RESEARCH; GAUCHER DISEASE; HEMATOLOGICAL PARAMETERS; HUMAN; MULTICENTER STUDY (TOPIC); PRIORITY JOURNAL; TREATMENT RESPONSE; CLINICAL TRIAL; DRUG ADMINISTRATION; FEMALE; MALE; MULTICENTER STUDY; PRESCHOOL CHILD; SEXUAL MATURATION; TREATMENT OUTCOME;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; DRUG ADMINISTRATION SCHEDULE; ENZYME REPLACEMENT THERAPY; FEMALE; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; HUMANS; MALE; SEXUAL MATURATION; TREATMENT OUTCOME;

EID: 84957842180     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2015.05.012     Document Type: Article

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