Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series-Reference-Cited by-同舟云学术

Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series

Published:2014-03-05 Issue:5 Volume:37 Page:823-829
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Lampe Christina,Bosserhoff Ann-Kathrin,Burton Barbara K.,Giugliani Roberto,de Souza Carolina F.,Bittar Camila,Muschol Nicole,Olson Rebecca,Mendelsohn Nancy J.

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://link.springer.com/content/pdf/10.1007/s10545-014-9686-7

Reference18 articles.

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3. Boado RJ, Hui EK, Lu JZ, Sumbria RK, Pardridge WM (2013) Blood–brain barrier molecular trojan horse enables imaging of brain uptake of radioiodinated recombinant protein in the rhesus monkey. Bioconjug Chem 24:1741–1749

4. Burton BK, Giugliani R (2012) Diagnosing Hunter syndrome in pediatric practice: practical considerations and common pitfalls. Eur J Pediatr 171:631–639

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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