A multicenter, open-label study evaluating safety and clinical outcomes in children (1.4-7.5 years) with Hunter syndrome receiving idursulfase enzyme replacement therapy

Genetics in Medicine

Volumn 16, Issue 6, 2014, Pages 435-441

  Giugliani, Roberto ^a,b   Hwu, Wuh Liang ^c   Tylki Szymanska, Anna ^d   Whiteman, David A H ^e   Pano, Arian ^e  

^a HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^b National Institute for Populational Medical Genetics   (Brazil)

^c NATIONAL TAIWAN UNIVERSITY HOSPITAL   (Taiwan)

^d CHILDREN'S MEMORIAL HEALTH INSTITUTE   (Poland)

^e Department of Shire ^*  (United States)

Author keywords

enzyme replacement therapycHunter syndrome; idursulfase; lysosomal storage disease; mucopolysaccharidosis II

Indexed keywords

GLYCOSAMINOGLYCAN; IDURONATE 2 SULFATASE; IMMUNOGLOBULIN G ANTIBODY; ANTI-IGG; ANTIIDIOTYPIC ANTIBODY;

ABDOMINAL PAIN; ARTICLE; BRONCHOPNEUMONIA; CHILD; CLINICAL ARTICLE; CYANOSIS; DENVER DEVELOPMENTAL SCREENING TEST; DIARRHEA; DRUG SAFETY; ELECTROCARDIOGRAM; EOSINOPHILIA; FEVER; FOLLOW UP; GASTROINTESTINAL INFECTION; GROWTH RATE; HEADACHE; HUMAN; HUNTER SYNDROME; HYPEREMIA; INFUSION RELATED REACTION; LABORATORY TEST; LIVER SIZE; MALAISE; MALE; MULTICENTER STUDY; NAUSEA; PHASE 4 CLINICAL TRIAL (TOPIC); PHYSICAL EXAMINATION; RASH; RESPIRATORY DISTRESS; SLEEP DISORDERED BREATHING; SPLEEN SIZE; URTICARIA; VITAL SIGN; VOMITING; CLINICAL TRIAL; DRUG EFFECTS; ENZYME REPLACEMENT; IMMUNOLOGY; INFANT; LIVER; MUCOPOLYSACCHARIDOSIS II; PRESCHOOL CHILD; SPLEEN; TREATMENT OUTCOME; URINE;

ANTIBODIES, ANTI-IDIOTYPIC; CHILD; CHILD, PRESCHOOL; ENZYME REPLACEMENT THERAPY; FOLLOW-UP STUDIES; GLYCOSAMINOGLYCANS; HUMANS; IDURONATE SULFATASE; INFANT; LIVER; MALE; MUCOPOLYSACCHARIDOSIS II; SPLEEN; TREATMENT OUTCOME;

EID: 84902073847     PISSN: 10983600     EISSN: 15300366     Source Type: Journal    
DOI: 10.1038/gim.2013.162     Document Type: Article

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