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Fabry Disease: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat: What Do We Know Today?

Fabry disease is a lysosomal storage disorder caused by the deficiency of the α-galactosidase-A enzyme. The result is the progressive accumulation of complex glycosphingolipids and cellular dysfunction. Cardiac, renal, and neurological involvement significantly reduces life expectancy. Currently, th...

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Detalles Bibliográficos
Autores principales:	Perretta, Fernando, Jaurretche, Sebastián
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Perspective
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9957019/ https://www.ncbi.nlm.nih.gov/pubmed/36832983 http://dx.doi.org/10.3390/healthcare11040449

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