Fabry Disease: The Current Treatment Landscape

Fabry disease (FD) is a rare X-linked lysosomal storage disease based on a deficiency of α-galactosidase A (AGAL) caused by mutations in the α-galactosidase A gene (GLA). The lysosomal accumulation of glycosphingolipids, especially globotriaosylceramide (Gb(3)) and globotriaosylsphingosine (lyso-Gb(...

Descripción completa

Detalles Bibliográficos
Autores principales: Lenders, Malte, Brand, Eva
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
Review Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8102455/
https://www.ncbi.nlm.nih.gov/pubmed/33721270
http://dx.doi.org/10.1007/s40265-021-01486-1

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8102455/
https://www.ncbi.nlm.nih.gov/pubmed/33721270
http://dx.doi.org/10.1007/s40265-021-01486-1

Ejemplares similares