Inherited deficiency in the mitochondrial protein frataxin causes the rare disease Friedreich's ataxia (FA), for which there is no successful treatment. We identified a redox deficiency in FA cells and used this to model the disease. We screened a 1600-compound library to identify existing drugs which could be of therapeutic benefit. We identified the topical anesthetic dyclonine as protective. Dyclonine increased FXN transcript and frataxin protein dose-dependently in FA cells and brains of animal models.

Read More: Dyclonine rescues frataxin deficiency in animal models and buccal cells of patients with Friedreich's Ataxia