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Safety outcomes and patients’ preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond

BACKGROUND: The Italian Medicines Agency (AIFA) demands precise information on benefit/risk profile of home-based enzyme replacement therapy (ERT) for the treatment of patients with Pompe disease and Mucopolysaccharidosis type I (MPS I). This passage is necessary to obtain the authorization for ERT...

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Detalles Bibliográficos
Autores principales:	Toscano, Antonio, Musumeci, Olimpia, Sacchini, Michele, Ravaglia, Sabrina, Siciliano, Gabriele, Fiumara, Agata, Verrecchia, Elena, Maione, Melania, Gentile, Jennifer, Fischetto, Rita, Crescimanno, Grazia, Taurisano, Roberta, Sechi, Annalisa, Gasperini, Serena, Cianci, Vittoria, Maggi, Lorenzo, Parini, Rossella, Lupica, Antonino, Scarpa, Maurizio
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2023
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10604412/ https://www.ncbi.nlm.nih.gov/pubmed/37891668 http://dx.doi.org/10.1186/s13023-023-02919-8

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