Enzyme Replacement Therapy for FABRY Disease: Possible Strategies to Improve Its Efficacy

Enzyme replacement therapy is the only therapeutic option for Fabry patients with completely absent AGAL activity. However, the treatment has side effects, is costly, and requires conspicuous amounts of recombinant human protein (rh-AGAL). Thus, its optimization would benefit patients and welfare/he...

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Detalles Bibliográficos
Autores principales: Iacobucci, Ilaria, Hay Mele, Bruno, Cozzolino, Flora, Monaco, Vittoria, Cimmaruta, Chiara, Monti, Maria, Andreotti, Giuseppina, Monticelli, Maria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Brief Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10003632/
https://www.ncbi.nlm.nih.gov/pubmed/36901983
http://dx.doi.org/10.3390/ijms24054548

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10003632/
https://www.ncbi.nlm.nih.gov/pubmed/36901983
http://dx.doi.org/10.3390/ijms24054548

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