Ontology highlight
ABSTRACT:
SUBMITTER: Zhang S
PROVIDER: S-EPMC6826337 | biostudies-literature | 2019 Apr
REPOSITORIES: biostudies-literature
Zhang Siyuan S Napierala Marek M Napierala Jill S JS
Trends in pharmacological sciences 20190401 4
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. ...[more]