Treatment of the neurodegenerative disease Friedreich´s ataxia

USE OF ERYTHROPOIETIN FOR THE TREATMENT OF
FRIEDREICH´S ATAXIA:
In addition to its neuro- and cardioprotective properties recombinant human erythropoietin (rhuEPO) significantly increases frataxin expression in primary lymphocytes from FApatients, and other cell types like neurons and primary human cardiac cells in vitro. (Sturm et.al 2005, Eur. J.Clin. Invest. 35(11):711-7) rhuEPO was successfully tested as a treatment for Friedreich`s ataxia in Phase IIa/IIb and dosefinding studies.

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