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EPI-743 Reverses the Progression of the Pediatric Mitochondrial Disease- Genetically Defined Leigh Syndrome

Genetically defined Leigh syndrome is a rare, fatal inherited neurodegenerative disorder that predominantly affects children. No treatment is available. EPI-743 is a novel small molecule developed for the treatment of Leigh syndrome and other inherited mitochondrial diseases. In compassionate use cases and in an FDA Expanded Access protocol, children with Leigh syndrome treated with EPI-743 demonstrated objective signs of neurologic and neuromuscular improvement.

Read More: EPI-743 Reverses the Progression of the Pediatric Mitochondrial Disease- Genetically Defined Leigh Syndrome

-Edison Pharma Press Release-


About the Author

Jen Farmer

Jen Farmer

Executive Director

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