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Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study

BACKGROUND: Pompe disease is a lysosomal storage disease treated with life-long enzyme replacement therapy (ERT). Home-based ERT has been provided in the Netherlands since 2008 because it diminishes the burden of treatment, increases patient flexibility and autonomy, and is thus a more patient-centr...

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Detalles Bibliográficos
Autores principales:	Ditters, Imke A. M., van der Beek, Nadine A. M. E., Brusse, Esther, van der Ploeg, Ans T., van den Hout, Johanna M. P., Huidekoper, Hidde H.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2023
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10169363/ https://www.ncbi.nlm.nih.gov/pubmed/37158969 http://dx.doi.org/10.1186/s13023-023-02715-4

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