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Mechanisms of Neutralizing Anti-drug Antibody Formation and Clinical Relevance on Therapeutic Efficacy of Enzyme Replacement Therapies in Fabry Disease
Fabry disease (FD) is a rare X-linked lysosomal storage disorder caused by mutations in the α-galactosidase A (AGAL/GLA) gene. The lysosomal accumulation of the substrates globotriaosylceramide (Gb(3)) and globotriaosylsphingosine (lyso-Gb(3)) results in progressive renal failure, cardiomyopathy ass...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Springer International Publishing
2021
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8602155/ https://www.ncbi.nlm.nih.gov/pubmed/34748189 http://dx.doi.org/10.1007/s40265-021-01621-y |