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Current and experimental therapeutics for Fabry disease
Fabry (or Anderson‐Fabry) is a rare pan‐ethnic disease affecting males and females. Fabry is an X‐linked lysosomal storage disease, affecting glycosphingolipid metabolism, that is caused by mutations of the GLA gene that codes for α‐galactosidase A. Fabry disease (FD) can be classified into a severe...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Blackwell Publishing Ltd
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8453747/ https://www.ncbi.nlm.nih.gov/pubmed/33997974 http://dx.doi.org/10.1111/cge.13999 |