Shortcast: Comparison of subcutaneous and intravenous moss-aGal in Fabry disease mouse model
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Enzyme Replacement Therapy (ERT) has changed the course of several lysosomal storage disorders but regular, intravenous administration is not without its issues. In this latest Shortcast, Dr Paulina Dabrowska-Schlepp describes her group's work to develop subcutaneous ERT for Fabry Disease.
Comparison of efficacy between subcutaneous and intravenous application of moss-aGal in the mouse model of Fabry disease
Paulina Dabrowska-Schlepp, et al
https://doi.org/10.1002/jmd2.12393
Shortcast: Comparison of subcutaneous and intravenous moss-aGal in Fabry disease mouse model