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A natural compound, designated as orphan drug for cystinuria

Cystine crystal aggregates present in the bladder of a mouse with cystinuria, observated at 500 magnifications in a scanning electron microscope. Image: Clara Mayayo, edited by Miguel López Heredia.

Cystine crystal aggregates present in the bladder of a mouse with cystinuria, observated at 500 magnifications in a scanning electron microscope. Image: Clara Mayayo, edited by Miguel López Heredia.

07/09/2021

A natural compound with antioxidant properties, L-ergothioneine, has been designed as an orphan drug for the treatment of cystinuria by the European Medicines Agency (EMA). The conducted studies showed in a murine model of this rare disease that the L-ergothioneine could prevent the formation of cystine stones in the kidney and the urinary tracts, common in this pathology, or delay its appearance. This orphan drug has been carried out by a team of the Bellvitge Institute for Biomedical Research (IDIBELL) and the University of Barcelona (UB), and has been promoted by the Rare Diseases Networking Biomedical Research Centre (CIBERER). The study has been led by Virginia Nunes, researcher at IDIBELL and professor at the Department of Physiological Sciences of the UB, and co-led by Miguel López de Heredia, researcher at CIBERER.

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