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Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report

BACKGROUND: Gaucher disease is one of the most common lipid-storage disorders, affecting approximately 1 in 75,000 births. Enzyme replacement therapy with recombinant glucocerebrosidase is currently considered the first-line treatment choice for patients with symptomatic Gaucher disease type 1. Oral...

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Detalles Bibliográficos
Autores principales:	Giuffrida, Gaetano, Lombardo, Rita, Di Francesco, Ernesto, Parrinello, Laura, Di Raimondo, Francesco, Fiumara, Agata
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2016
Materias:	Case Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5100336/ https://www.ncbi.nlm.nih.gov/pubmed/27821156 http://dx.doi.org/10.1186/s13256-016-1060-y

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