Therapeutic Prospects for Friedreich's Ataxia

Academic Article

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.
  • Published In

    Digital Object Identifier (doi)

    Author List

  • Zhang S; Napierala M; Napierala JS
  • Start Page

  • 229
  • End Page

  • 233
  • Volume

  • 40
  • Issue

  • 4