Omaveloxolone and Beyond: Emerging Treatments in Friedreich’s...

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2025-06-17 11:46:06

Omaveloxolone and Beyond: Emerging Treatments in Friedreich’s Ataxia

Friedreich’s Ataxia (FA) is a rare, autosomal recessive neurodegenerative disorder that primarily affects the nervous system and the heart. Caused by mutations in the FXN gene, FA leads to decreased production of frataxin, a mitochondrial protein essential for iron-sulfur cluster formation and mitochondrial energy production. Its deficiency results in mitochondrial dysfunction, oxidative...

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