A dose-optimization trial of laronidase (Aldurazyme®) in patients with mucopolysaccharidosis I

Molecular Genetics and Metabolism

Volumn 96, Issue 1, 2009, Pages 13-19

  Giugliani, Roberto ^a   Rojas, Verónica Muñoz ^a   Martins, Ana Maria ^b   Valadares, Eugênia R ^c   Clarke, Joe T R ^d   Góes, José E C ^e   Kakkis, Emil D ^f   Worden, Mary Alice ^g   Sidman, Marisa ^g   Cox, Gerald F ^g,h  

^a FEDERAL UNIVERSITY OF RIO GRANDE DO SUL   (Brazil)

^b FEDERAL UNIVERSITY OF SÃO PAULO   (Brazil)

^c FEDERAL UNIVERSITY OF MINAS GERAIS   (Brazil)

^d HOSPITAL FOR SICK CHILDREN   (Canada)

^e HOSPITAL INFANTIL JOANA DE GUSMÃO   (Brazil)

^f BIOMARIN PHARMACEUTICAL INC   (United States)

^g GENZYME CORPORATION   (United States)

^h BOSTON CHILDREN S HOSPITAL   (United States)

Author keywords

Clinical trial; Dose optimization; Enzyme replacement therapy; Glycosaminoglycans; Hurler; Hurler Scheie; Laronidase; Mucopolysaccharidosis (MPS) type I; Scheie

Indexed keywords

ANTIHISTAMINIC AGENT; ANTIPYRETIC AGENT; FENOTEROL; GLYCOSAMINOGLYCAN; LARONIDASE;

ACUTE RESPIRATORY FAILURE; ADOLESCENT; ADULT; ARTICLE; BRONCHITIS; BRONCHODILATATION; CHILD; CLINICAL ARTICLE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CYANOSIS; DIARRHEA; DRUG DOSE COMPARISON; DRUG DOSE REGIMEN; DRUG EFFECT; DRUG ERUPTION; DRUG INFUSION; DRUG SAFETY; ENZYME REPLACEMENT; ERYTHEMA; FEMALE; FEVER; HUMAN; HURLER SYNDROME; INJECTION SITE REACTION; INSOMNIA; LEUKOCYTE DISORDER; LIVER WEIGHT; MALE; MULTICENTER STUDY; OPTIMAL DRUG DOSE; PALLOR; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN URINE LEVEL; RANDOMIZED CONTROLLED TRIAL; RISK BENEFIT ANALYSIS; SCHOOL CHILD; SEROCONVERSION; SIDE EFFECT; THERAPY EFFECT; THROMBOCYTE COUNT; TREATMENT OUTCOME; TREATMENT RESPONSE; URINARY EXCRETION; URTICARIA; VOMITING;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; DOSE-RESPONSE RELATIONSHIP, DRUG; DRUG ADMINISTRATION SCHEDULE; FEMALE; GLYCOSAMINOGLYCANS; HUMANS; IDURONIDASE; INFUSIONS, INTRAVENOUS; MALE; MUCOPOLYSACCHARIDOSIS I; YOUNG ADULT;

EID: 57649092465     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2008.10.009     Document Type: Article

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