University of Pennsylvania

The Children's Hospital of Philadelphia

Maria Grazia Cotticelli

Donna Mary Huryn

Robert B Wilson

Amos Smith

Phillip A Benedetti

Jason E Melvin

Shujuan Xia

Roberto Forestieri

Taehee Lee

The invention relates to compounds of Formula I or Formula II: to compositions comprising such compounds, and to methods of use thereof for treating neurodegenerative disorders, such as Friedreich ataxia.

Oleic acid derivatives as treatments for Friedreich ataxia and inhibitors of ferroptosis

Methods for improving mitochondrial function, decreasing iron accumulation, and/or decreasing oxidative stress by exposing cells or treating a subject to compounds or compositions of the general formula are described that are beneficial in treating, for example, diseases and conditions such as Friedreich's ataxia, normal aging, and various neurodegenerative diseases such as Alzheimer's disease and Parkinson's disease. Furthermore, such compounds are useful as probes for identifying defects in mitochondrial metabolism, mitochondrial iron accumulation, cellular stress among other mitochondrial diseases and helping to identify compounds active in overcoming such defects.

Small molecule activators of mitochondrial function

Methods for improving mitochondrial function, decreasing iron accumulation, and/or decreasing oxidative stress by exposing cells or treating a subject to compounds or compositions of the general formula (I) are described that are beneficial in treating, for example, diseases and conditions such as Friedreich's ataxia, normal aging, and various neurodegenerative diseases such as Alzheimer's disease and Parkinson's disease. Furthermore, such compounds are useful as probes for identifying defects in mitochondrial metabolism, mitochondrial iron accumulation, cellular stress among other mitochondrial diseases and helping to identify compounds active in overcoming such defects. 

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Philadelphia, PA, United States of America

Maria Grazia Cotticelli