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Moving Towards New Therapies for Fabry Disease

Susanne Seemann M Ernst Chiara Cimmaruta Stephan Struckmann Dr. rer. nat. Claudia Cozma, MD Dirk Koczan Anne-Marie Knospe LR Haake Valentina Citro Anja Bräuer, PhD Giuseppina Andreotti Maria Cubellis Georg Fuellen Andreas Hermann, MD Anne Katrin Giese, MD Prof. Arndt Rolfs, MD Jan Lukas, PhD
January 03, 2020

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.