Pharmacological therapeutics in Friedreich ataxia: the present state.

  title={Pharmacological therapeutics in Friedreich ataxia: the present state.},
  author={Cassandra J Strawser and Kimberly A Schadt and Lauren A. Hauser and Ashley J McCormick and McKenzie Wells and Jane Larkindale and Hong Lin and David R Lynch},
  journal={Expert review of neurotherapeutics},
  volume={17 9},
INTRODUCTION Friedreich ataxia (FRDA) is a progressive, inherited, neurodegenerative disease for which there is currently no cure or approved treatment. FRDA is caused by deficits in the production and expression of frataxin, a protein found in the mitochondria that is most likely responsible for regulating iron-sulfur cluster enzymes within the cell. A… CONTINUE READING