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Current and Investigational Therapeutics for Fabry Disease
Fabry disease (FD) is an X-linked lysosomal storage disease caused by a deficiency in the lysosomal enzyme α-galactosidase (α-GAL). This in turn leads to the buildup of globotriaosylceramide, resulting classically in progressive kidney disease, peripheral neuropathy, early-onset cerebrovascular dise...
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| Publicado no: | Kidney Int Rep |
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| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Elsevier
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7136345/ https://ncbi.nlm.nih.gov/pubmed/32274449 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ekir.2019.11.013 |
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