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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Detalles Bibliográficos
Autores principales: Lampe, Christina, Bosserhoff, Ann-Kathrin, Burton, Barbara K., Giugliani, Roberto, de Souza, Carolina F., Bittar, Camila, Muschol, Nicole, Olson, Rebecca, Mendelsohn, Nancy J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Netherlands 2014
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4158409/
https://www.ncbi.nlm.nih.gov/pubmed/24596019
http://dx.doi.org/10.1007/s10545-014-9686-7

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