Therapeutic Prospects for Friedreich's Ataxia.

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.

SUBMITTER: Zhang S 

PROVIDER: S-EPMC6826337 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature