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Renoprotective Effect of Agalsidase Alfa: A Long-Term Follow-Up of Patients with Fabry Disease
Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene, which, without treatment, can cause significant renal dysfunction. We evaluated the effects of enzyme replacement therapy with agalsidase alfa on renal decline in patients with Fabry disease using data from the F...
Autores principales: | Cybulla, Markus, Nicholls, Kathleen, Feriozzi, Sandro, Linhart, Aleš, Torras, Joan, Vujkovac, Bojan, Botha, Jaco, Anagnostopoulou, Christina, West, Michael L. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9410255/ https://www.ncbi.nlm.nih.gov/pubmed/36013057 http://dx.doi.org/10.3390/jcm11164810 |
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