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Article

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

International journal of molecular sciences - 4 May 2022

Monticelli Maria, Liguori Ludovica, Allocca Mariateresa, Bosso Andrea, Andreotti Giuseppina, Lukas Jan, Monti Maria Chiara, Morretta Elva, Cubellis Maria Vittoria, Hay Mele Bruno

Abstract excerpt

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 reversible inhibitor of alpha-galactosidase. We looked for molecules that can potentiate this pharmacological chaperone,...

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Identifiers

PMID: 35563496
DOI: 10.3390/ijms23095105
PMCID: PMC9105905

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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