Breakthroughs in Friedreich’s Ataxia Treatment: From Hope to...

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2026-01-19 05:29:34 -

Breakthroughs in Friedreich’s Ataxia Treatment: From Hope to Reality

1. 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...

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