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Mutations in Mitochondrial Genes Ameliorate Pathology in Friedreich’s Ataxia Model

Scientists from Mass General Brigham and the Broad Institute have been studying more effective ways of treating people living with Friedreich’s ataxia (FA). People with this rare and progressive neuromuscular disorder are often diagnosed between the ages of five and 15 and live into their 30s or 40s. A key challenge is that there are no widely approved treatments for the disease, and existing therapies may not be effective in all cases. But that…

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GEN - Genetic Engineering and Biotechnology News… broke the news in on Thursday, December 11, 2025.
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