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FARAFARA Cure FA

Epigenetic therapy for Friedreich's ataxia

OBJECTIVE:

To investigate whether a histone deacetylase inhibitor (HDACi) would be effective in an in vitro model for the neurodegenerative disease Friedreich ataxia (FRDA) and to evaluate safety and surrogate markers of efficacy in a phase I clinical trial in patients.

Read More: Epigenetic therapy for Friedreich's ataxia

About the Author

Jen Farmer

Jen Farmer

Executive Director

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