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

INTRODUCTION: No published clinical trial data are available to inform the use of enzyme replacement therapy (ERT) in patients with the severe (neuropathic) phenotype of mucopolysaccharidosis II (MPS II). Current guidelines recommend ERT administered intravenously be used on a trial basis in this po...

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Библиографические подробности
Главные авторы: Lampe, Christina, Bosserhoff, Ann-Kathrin, Burton, Barbara K., Giugliani, Roberto, de Souza, Carolina F., Bittar, Camila, Muschol, Nicole, Olson, Rebecca, Mendelsohn, Nancy J.
Формат: Artigo
Язык:Inglês
Опубликовано: Springer Netherlands 2014
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Original Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4158409/
https://ncbi.nlm.nih.gov/pubmed/24596019
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-014-9686-7
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