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Drug Repositioning for Fabry Disease: Acetylsalicylic Acid Potentiates the Stabilization of Lysosomal Alpha-Galactosidase by Pharmacological Chaperones

Fabry disease is caused by a deficiency of lysosomal alpha galactosidase and has a very large genotypic and phenotypic spectrum. Some patients who carry hypomorphic mutations can benefit from oral therapy with a pharmacological chaperone. The drug requires a very precise regimen because it is a reve...

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Detalles Bibliográficos
Autores principales: Monticelli, Maria, Liguori, Ludovica, Allocca, Mariateresa, Bosso, Andrea, Andreotti, Giuseppina, Lukas, Jan, Monti, Maria Chiara, Morretta, Elva, Cubellis, Maria Vittoria, Hay Mele, Bruno
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9105905/
https://www.ncbi.nlm.nih.gov/pubmed/35563496
http://dx.doi.org/10.3390/ijms23095105

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