Abstract
Friedreich's ataxia (FA) is a recessive ataxia caused by reduction of mitochondrial protein, frataxin (FXN). Cardiomyopathy is the leading cause of death in FA patients due to deficient FXN expression in the heart. We have developed a novel monomethyl fumarate prodrug, IMF, which has improved pharmacokinetic profile and compared its effect to fumarate prodrug dimethyl fumarate (DMF, Tecfidera).
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