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Therapeutic Prospects for Friedreich's Ataxia
Siyuan Zhang1, Marek Napierala1, Jill S Napierala1
1Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham (UAB), 1825 University Boulevard, Birmingham, AL 35294, USA.
Trends in Pharmacological Sciences
|March 26, 2019
Abstract:
Friedreich's ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or alleviate the consequences of frataxin deficiency. Recent significant advances in the FRDA therapeutic pipeline are bringing patients closer to a cure.