Early experience on omaveloxolone in adult patients with Friedreich's ataxia: a real-world observational study
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Introduction: Friedreich's ataxia (FRDA) is an autosomal recessive neurodegenerative spinocerebellar ataxia caused by a homozygous GAA triplet repeat expansion in the frataxin (FXN) gene. FRDA is a multisystem disorder involving... ...
