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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...

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Detalles Bibliográficos
Autores principales: Lenders, Malte, Brand, Eva
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
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