Emerging Therapies in Friedreich's Ataxia: A Review

Friedreich's ataxia (FRDA) is an inherited, neurodegenerative disease that typically presents in childhood and results progressive gait limb ataxia, with the extraneural features of hypertrophic cardiomyopathy, diabetes scoliosis. The genetic defect a deficiency frataxin protein, which important for mitochondrial function, especially brain heart. Drug development has approached FRDA through pathways addressing oxidative stress, dysfunction, protein DNA transcriptional deficiency, paving way first disease-modifying drugs FRDA.