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).
Monday, February 13, 2023
Bioactive fumarate improves cardiac function and expands lifespan in Friedrech’s ataxia
Salinas, L., Figueroa, F., Montgomery, C. B., Thai, P. N., Chiamvimonvat, N., Dugar, S., Sen, S., Cortopassi, G., & Dedkova, E. N. (2023). Bioactive fumarate improves cardiac function and expands lifespan in Friedrech’s ataxia. Biophysical Journal, 122(3), 96a. doi:10.1016/j.bpj.2022.11.714
