The role of enzyme replacement therapy in severe Hunter syndrome—an expert panel consensus-Reference-Cited by-同舟云学术

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

Published:2011-10-29 Issue:1 Volume:171 Page:181-188
ISSN:0340-6199
Container-title:European Journal of Pediatrics
language:en
Short-container-title:Eur J Pediatr

Author:

Muenzer Joseph,Bodamer Olaf,Burton Barbara,Clarke Lorne,Frenking Gudrun Schulze,Giugliani Roberto,Jones Simon,Rojas Maria Verónica Muñoz,Scarpa Maurizio,Beck Michael,Harmatz Paul

Publisher

Springer Science and Business Media LLC

Subject

Pediatrics, Perinatology, and Child Health

Link

http://www.springerlink.com/index/pdf/10.1007/s00431-011-1606-3

Reference42 articles.

1. Bach G, Eisenberg F Jr, Cantz M, Neufeld EF (1973) The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase. Proc Natl Acad Sci U S A 70:2134–2138

2. Bagewadi S, Roberts J, Mercer J, Jones S, Stephenson J, Wraith JE (2008) Home treatment with Elaprase and Naglazyme is safe in patients with mucopolysaccharidoses types II and VI, respectively. J Inherit Metab Dis 31:733–737

3. Burton BK, Guffon N, Roberts J, van der Ploeg AT, Jones SA, HOS investigators (2010) Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II—data from the Hunter Outcome Survey. Mol Genet Metab 101:123–129

4. Burton BK, Wiesman C, Paras A, Kim K, Katz R (2009) Home infusion therapy is safe and enhances compliance in patients with mucopolysaccharidoses. Mol Genet Metab 97:234–236

5. Coman DJ, Hayes IM, Collins V, Sahhar M, Wraith JE, Delatycki MB (2008) Enzyme replacement therapy for mucopolysaccharidoses: opinions of patients and families. J Pediatr 152:723–727

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