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Source: Children's Hospital of Philadelphia  Feb 27, 2019  5 years, 1 month, 2 weeks, 6 days, 14 hours, 28 minutes ago

Cysteamine Bitartrate may have therapeutic potential in severe energy deficiency and mitochondrial disease

Cysteamine Bitartrate may have therapeutic potential in severe energy deficiency and mitochondrial disease
Source: Children's Hospital of Philadelphia  Feb 27, 2019  5 years, 1 month, 2 weeks, 6 days, 14 hours, 28 minutes ago
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.
cysteamine-bitartrate

"We found therapeutic potential in mitochondrial disease, based on evidence for neuroprotection in two different animal models," said Marni J. Falk, MD, at Children's Hospital of Philadelphia (CHOP), the Executive Director of CHOP's Mitochondrial Medicine Frontier Program. "However, we also showed that this drug has a narrow therapeutic window -- even small increases in dosage were toxic in diverse laboratory animals and human cells. This implies t