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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...
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 |
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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/PMC9105905/ https://www.ncbi.nlm.nih.gov/pubmed/35563496 http://dx.doi.org/10.3390/ijms23095105 |
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