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Home infusion with Elosulfase alpha (Vimizim(R)) in a UK Paediatric setting

Enzyme replacement therapy is the only available treatment for Mucopolysaccharidosis type IVA (MPS IVA, Morquio syndrome). The treatment is lengthy and invasive involving weekly intravenous infusions of 4–5 h. This can cause significant disruption to normal family life so the provision of a safe and...

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Detalles Bibliográficos
Autores principales:	Finnigan, Niamh, Roberts, Jane, Mercer, Jean, Jones, Simon A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2017
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5675729/ https://www.ncbi.nlm.nih.gov/pubmed/29159073 http://dx.doi.org/10.1016/j.ymgmr.2017.10.012

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