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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(...
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| Publicado no: | Drugs |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Springer International Publishing
2021
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8102455/ https://ncbi.nlm.nih.gov/pubmed/33721270 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40265-021-01486-1 |
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