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Enzyme replacement therapy in two patients with classic Fabry disease from the same family tree: Two case reports
BACKGROUND: The pathophysiology of Fabry disease (FD)-induced progressive vital organ damage is irreversible. Disease progression can be delayed using enzyme replacement therapy (ERT). In patients with classic FD, sporadic accumulation of globotriaosylceramide (GL-3) in the heart and kidney begins i...
| Autores principales: | , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Baishideng Publishing Group Inc
2023
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10294186/ https://www.ncbi.nlm.nih.gov/pubmed/37383915 http://dx.doi.org/10.12998/wjcc.v11.i15.3542 |