Researchers find effective combination of therapies for managing mitochondrial disease

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Study is first to demonstrate how one specific combinational therapy may provide measurable benefits to patients with mitochondrial respiratory chain disorders, which currently lack FDA-approved therapies


Philadelphia, March 2, 2021 – Researchers from the Mitochondrial Medicine Frontier Program at Children’s Hospital of Philadelphia (CHOP) have demonstrated how one combination of therapies may be beneficial for patients with mitochondrial respiratory chain disorders. This preclinical research paves the way to develop more tailored treatment options for patients with inherited mitochondrial disease and acquired energy disorders. The findings emphasize the importance of rational therapeutic modeling to target specific cellular deficiencies and provide proper cellular nutrition as an effective means to manage mitochondrial disease.

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