CRIM-negative infantile Pompe disease: Characterization of immune responses in patients treated with ERT monotherapy

Genetics in Medicine

Volumn 17, Issue 11, 2015, Pages 912-918

  Berrier, Kathryn L ^a   Kazi, Zoheb B ^a   Prater, Sean N ^a   Bali, Deeksha S ^a   Goldstein, Jennifer ^a   Stefanescu, Mihaela C ^a   Rehder, Catherine W ^b   Botha, Eleanor G ^c   Ellaway, Carolyn ^d   Bhattacharya, Kaustuv ^d   Tylki Szymanska, Anna ^e   Karabul, Nesrin ^f   Rosenburg, Amy S ^g   Kishnani, Priya S ^a  

^a DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b DUKE UNIVERSITY MEDICAL CENTER   (United States)

^c EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

^e CHILDREN'S MEMORIAL HEALTH INSTITUTE   (Poland)

^f UNIVERSITY MEDICAL CENTER MAINZ   (Germany)

^g CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

Alglucosidase alfa; antibodies; enzyme replacement therapy; immune tolerance; Pompe disease

Indexed keywords

RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; ALLOANTIBODY; ALPHA GLUCOSIDASE; IMMUNOGLOBULIN G;

ANTIBODY TITER; ARTICLE; CROSS REACTIVE IMMUNOLOGIC MATERIAL NEGATIVE INFANTILE POMPE DISEASE; ENZYME REPLACEMENT; FEMALE; FRAMESHIFT MUTATION; GLYCOGEN STORAGE DISEASE TYPE 2; HEART LEFT VENTRICLE MASS; HUMAN; IMMUNE RESPONSE; IMMUNOMODULATION; INFANT; INFANT DISEASE; MAJOR CLINICAL STUDY; MALE; MEDICAL RECORD REVIEW; MONOTHERAPY; NEWBORN; NONSENSE MUTATION; OVERALL SURVIVAL; TREATMENT OUTCOME; ADVERSE EFFECTS; BLOOD; CROSS REACTION; GENETICS; GLYCOGEN STORAGE DISEASE TYPE II; IMMUNOLOGY; KAPLAN MEIER METHOD; MORTALITY; MUTATION;

ALPHA-GLUCOSIDASES; CROSS REACTIONS; ENZYME REPLACEMENT THERAPY; FEMALE; GLYCOGEN STORAGE DISEASE TYPE II; HUMANS; IMMUNOGLOBULIN G; INFANT; INFANT, NEWBORN; ISOANTIBODIES; KAPLAN-MEIER ESTIMATE; MALE; MUTATION; TREATMENT OUTCOME;

EID: 84946607678     PISSN: 10983600     EISSN: 15300366     Source Type: Journal    
DOI: 10.1038/gim.2015.6     Document Type: Article

References (20)

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