Lessons for clinical trial design in Friedreich's ataxia

Masha G Savelieff Eva L Feldman The Lancet Neurology; Published:March 23, 2021DOI:https://doi.org/10.1016/S1474-4422(21)00064-8 Friedreich's ataxia is a rare autosomal-recessive disease caused by mutations in the FXN gene, which encodes frataxin, a mitochondrial protein.  It is the most common inherited ataxia, which usually manifests as gait unsteadiness in adolescence, with slowly progressive trunk and limb ataxia, and eventual loss of independent movement.  Friedreich's ataxia often involves the heart and musculoskeletal system and raises diabetes risk, making it a multisystem disorder.