Comparison of efficacy between subcutaneous and intravenous application of moss‐aGal in the mouse model of Fabry disease

Fabry disease (FD, OMIM 301500) is a rare X‐linked inherited lysosomal storage disorder associated with reduced activities of α‐galactosidase A (aGal, EC 3.2.1.22). The current standard of care for FD is based on enzyme replacement therapy (ERT), in which a recombinantly produced version of αGal is...

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Detalles Bibliográficos
Autores principales: Dabrowska‐Schlepp, Paulina, Busch, Andreas, Shen, Jin‐Song, Cheong, Rachel Y., Madsen, Lone Bruhn, Mascher, Daniel, Schiffmann, Raphael, Schaaf, Andreas
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2023
Materias:
Research Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10623099/
https://www.ncbi.nlm.nih.gov/pubmed/37927484
http://dx.doi.org/10.1002/jmd2.12393

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10623099/
https://www.ncbi.nlm.nih.gov/pubmed/37927484
http://dx.doi.org/10.1002/jmd2.12393

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