TY  - JOUR
AU  - Besada, Pedro
AU  - Gallardo-Gomez, Maria
AU  - Perez-Marquez, Tania
AU  - Patino-alvarez, Lucia
AU  - Pantano, Sergio
AU  - Silva-Lopez, Carlos
AU  - Teran, Carmen
AU  - Arevalo-Gomez, Ana
AU  - Ruz-Zafra, Aurora
AU  - Fernandez-Martin, Julian
AU  - Ortolano, Saida
PY  - 2021
DO  - 10.3390/biom11121856
UR  - https://hdl.handle.net/10668/24783
T2  - Biomolecules
AB  - Fabry disease is an X-linked multisystemic disorder caused by the impairment of lysosomal alpha-Galactosidase A, which leads to the progressive accumulation of glycosphingolipids and to defective lysosomal metabolism. Currently, Fabry disease is...
LA  - en
PB  - Mdpi
KW  - Fabry disease
KW  - pharmacological chaperones
KW  - GLA variants
KW  - Migalastat
KW  - lysosomal storage diseases
KW  - Therapy
KW  - Defect
TI  - The New Pharmacological Chaperones PBXs Increase alpha-Galactosidase A Activity in Fabry Disease Cellular Models
TY  - research article
VL  - 11
ER  -