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FARAFARA Cure FA

Epigenetic-based therapies for Friedreich ataxia

Friedreich ataxia (FRDA) is a lethal autosomal recessive neurodegenerative disorder caused primarily by a homozygous GAA repeat expansion mutation within the first intron of the FXN gene, leading to inhibition of FXN transcription and thus reduced frataxin protein expression.

Read More: Epigenetic-based therapies for Friedreich ataxia


About the Author

Jen Farmer

Jen Farmer

Executive Director

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