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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Main Authors: Lampe, Christina, Bosserhoff, Ann-Kathrin, Burton, Barbara K., Giugliani, Roberto, de Souza, Carolina F., Bittar, Camila, Muschol, Nicole, Olson, Rebecca, Mendelsohn, Nancy J.
格式: Artigo
語言:Inglês
出版: Springer Netherlands 2014
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Original Article
在線閱讀: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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