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ARTICLE | Translation in Brief

Ciclopirox the chaperone

How ciclopirox may get another life as treatment for Günther disease

October 17, 2018 8:38 PM UTC

Atlas Molecular Pharma S.L. plans to repurpose antifungal drug ciclopirox to treat congenital erythropoietic porphyria after discovering it allosterically stabilizes an enzyme driving the disease. Atlas and colleagues from Center for Cooperative Research in Biosciences (CIC bioGUNE) described the mechanism in a Science Translational Medicine study published last month.

There are no approved therapies for congenital erythropoietic porphyria (Günther disease), in which missense mutations cause an enzyme in the heme biosynthetic pathway called uroporphyrinogen III synthase (UROS) to fold less efficiently. The two most common mutations are C73R and P248Q. ...