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The New Pharmacological Chaperones PBXs Increase α-Galactosidase A Activity in Fabry Disease Cellular Models

Fabry disease is an X-linked multisystemic disorder caused by the impairment of lysosomal α-Galactosidase A, which leads to the progressive accumulation of glycosphingolipids and to defective lysosomal metabolism. Currently, Fabry disease is treated by enzyme replacement therapy or the orally admini...

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Hoofdauteurs: Pedro Besada, María Gallardo-Gómez, Tania Pérez-Márquez, Lucía Patiño-Álvarez, Sergio Pantano, Carlos Silva-López, Carmen Terán, Ana Arévalo-Gómez, Aurora Ruz-Zafra, Julián Fernández-Martín, Saida Ortolano
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: MDPI AG 2021-12-01
Reeks:Biomolecules
Onderwerpen:
Fabry disease
pharmacological chaperones
<i>GLA</i> variants
Migalastat
lysosomal storage diseases
Online toegang:https://www.mdpi.com/2218-273X/11/12/1856
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