The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus

European Journal of Pediatrics

Volumn 171, Issue 1, 2012, Pages 181-188

  Muenzer, Joseph ^a   Bodamer, Olaf ^b   Burton, Barbara ^c   Clarke, Lorne ^d   Frenking, Gudrun Schulze ^e   Giugliani, Roberto ^f   Jones, Simon ^g   Rojas, Maria Verónica Muñoz ^h   Scarpa, Maurizio ⁱ   Beck, Michael ^e   Harmatz, Paul ^j  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF MIAMI MILLER SCHOOL OF MEDICINE   (United States)

^c NORTHWESTERN UNIVERSITY   (United States)

^d UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^e JOHANNES GUTENBERG UNIVERSITY   (Germany)

^f INAGEMP ^*  (Brazil)

^g ST MARY S HOSPITAL   (United Kingdom)

^h Medical Department   (Brazil)

ⁱ UNIVERSITY OF PADOVA   (Italy)

^j CHILDREN S HOSPITAL OAKLAND   (United States)

more..

Author keywords

Cognitive impairment; Enzyme replacement therapy; Hunter syndrome; Idursulfase; Mucopolysaccharidosis II; Severe phenotype

Indexed keywords

GLYCOSAMINOGLYCAN; IDURONATE 2 SULFATASE;

ARTICLE; BLOOD BRAIN BARRIER; COGNITION; DISEASE SEVERITY; ENZYME REPLACEMENT; FORCED VITAL CAPACITY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; PHENOTYPE; PRIORITY JOURNAL;

DISEASE PROGRESSION; ENZYME REPLACEMENT THERAPY; HUMANS; IDURONATE SULFATASE; MUCOPOLYSACCHARIDOSIS II; PHENOTYPE; PRACTICE GUIDELINES AS TOPIC; TREATMENT OUTCOME;

EID: 84857061064     PISSN: 03406199     EISSN: 14321076     Source Type: Journal    
DOI: 10.1007/s00431-011-1606-3     Document Type: Article

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