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3.2 Syndromes caused by expansion of DNA 3.2.1 Friedreich’s ataxia Friedreich ataxia (FRDA) is a rare autosomal-recessive neurodegenerative and cardiac disorder characterized by progressive limb and gait ataxia, dysarthria, dysphagia, loss of deep tendon reflexes, oculomotor dysfunction, and cardiomyopathy, with additional symptoms like diabetes mellitus and defective hearing in some cases

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At Week 4, most subjects were rated as having mild or moderate improvement (75%), while no subjects showed marked improvement