Fabry Disease Therapy: State-of-the-Art and Current Challenges

Fabry disease (FD) is a lysosomal storage disorder caused by mutations of the GLA gene that lead to a deficiency of the enzymatic activity of α-galactosidase A. Available therapies for FD include enzyme replacement therapy (ERT) (agalsidase alfa and agalsidase beta) and the chaperone migalastat. Des...

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Detalles Bibliográficos
Autores principales: Azevedo, Olga, Gago, Miguel Fernandes, Miltenberger-Miltenyi, Gabriel, Sousa, Nuno, Cunha, Damião
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7794923/
https://www.ncbi.nlm.nih.gov/pubmed/33379210
http://dx.doi.org/10.3390/ijms22010206

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