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Breakthroughs in Friedreich’s Ataxia Treatment: From Hope to Reality1. Introduction: Friedreich’s Ataxia Friedreich’s ataxia (FRDA) is a rare inherited neurodegenerative disorder that affects the nervous system and heart. It is caused by a GAA trinucleotide repeat expansion in the FXN gene on chromosome 9, leading to reduced production of frataxin, a mitochondrial protein vital for iron–sulfur (Fe–S) cluster formation. This deficiency...0 Commentarios 0 Acciones 12 Views 0 Vista previaPlease log in to like, share and comment!
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