From Genetic Mutation to Therapy in Friedreich Ataxia: Molecular Mechanisms, Therapeutic Advances, and Translational Challenges

Osaki, Y.; Haque, U. S.; Yokota, T. From Genetic Mutation to Therapy in Friedreich Ataxia: Molecular Mechanisms, Therapeutic Advances, and Translational Challenges. Preprints 2026, 2026041108. doi: 10.20944/preprints202604.1108.v1 

This review provides a comprehensive overview of the evolving therapeutic landscape in FRDA, highlighting mechanistic rationales, preclinical progress, clinical trial outcomes, and the key translational challenges that must be addressed to achieve durable disease modification.

From Genetic Mutation to Therapy in Friedreich Ataxia: Molecular Mechanisms, Therapeutic Advances, and Translational Challenges