Moving Towards New Therapies for Fabry Disease
Proteostasis regulators modulate proteasomal activity and gene expression to attenuate multiple phenotypes in Fabry disease
Fabry disease is a multi-system lysosomal storage disorder. As it is caused by an enzyme deficiency, enzyme replacement therapy is the standard treatment option. To overcome some of the issues with this strategy, alternatives are being explored through a collaborative effort involving researchers from CENTOGENE. The recent finding of proteostasis regulators representing a promising therapeutic target was published in the Biochemical Journal.