Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome)

Orphanet Journal of Rare Diseases

Volumn 10, Issue 1, 2015, Pages

  Pano, Arian ^a   Barbier, Ann J ^a   Bielefeld, Bonnie ^a   Whiteman, David AH ^a   Amato, David A ^a,b  

^a SHIRE   (United States)

^b VERTEX PHARMACEUTICALS   (United States)

Author keywords

Antibodies; Idursulfase; Mucopolysaccharidosis II; Treatment outcome

Indexed keywords

GLYCOSAMINOGLYCAN; IDURONATE 2 SULFATASE; IMMUNOGLOBULIN G ANTIBODY; NEUTRALIZING ANTIBODY; IMMUNOGLOBULIN G;

ALTERNATIVE RNA SPLICING; ARTICLE; CHILD; CLINICAL ARTICLE; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; FRAMESHIFT MUTATION; GENE DELETION; GENE REARRANGEMENT; GENOTYPE; HUMAN; HUNTER SYNDROME; IMMUNOGENICITY; INFUSION RELATED REACTION; LIVER SIZE; MALE; MISSENSE MUTATION; NONSENSE MUTATION; OPEN STUDY; OUTCOME ASSESSMENT; POST HOC ANALYSIS; RESPIRATORY TRACT INFECTION; SEROCONVERSION; SPLEEN SIZE; STATISTICAL SIGNIFICANCE; TREATMENT DURATION; TREATMENT RESPONSE; BLOOD; CLINICAL TRIAL; ENZYME REPLACEMENT; IMMUNOLOGY; INFANT; LIVER; MUCOPOLYSACCHARIDOSIS II; ORGAN SIZE; PATHOLOGY; PRESCHOOL CHILD; RISK FACTOR; SPLEEN; TREATMENT OUTCOME;

CHILD; CHILD, PRESCHOOL; ENZYME REPLACEMENT THERAPY; GENOTYPE; HUMANS; IDURONATE SULFATASE; IMMUNOGLOBULIN G; INFANT; LIVER; MALE; MUCOPOLYSACCHARIDOSIS II; ORGAN SIZE; RISK FACTORS; SPLEEN; TREATMENT OUTCOME;

EID: 85016650277     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-015-0265-2     Document Type: Article

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