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03/07/2019

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.


Source: https://www.sciencedaily.com/releases/2019/02/190226125014.htm

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



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