Memarian e_Learning Institute



Back to overview


An existing drug may have therapeutic potential in mitochondrial disease

New preclinical findings from extensive cell and animal studies suggest that a drug already used for a rare kidney disease could benefit patients with some mitochondrial disorders -- complex conditions with severe energy deficiency for which no proven effective treatments exist. Future clinical research is needed to explore whether the drug, cysteamine bitartrate, will meaningfully benefit patients.


Sujay Guha, Chigoziri Konkwo, Manuela Lavorato, Neal D Mathew, Min Peng, Julian Ostrovsky, Young-Joon Kwon, Erzsebet Polyak, Richard Lightfoot, Christoph Seiler, Rui Xiao, Michael Bennett, Zhe Zhang, Eiko Nakamaru-Ogiso, Marni J Falk. Pre-clinical evaluation of cysteamine bitartrate as a therapeutic agent for mitochondrial respiratory chain disease. Human Molecular Genetics, 2019; DOI: 10.1093/hmg/ddz023

Back to overview