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

Internet

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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