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Edison Pharmaceuticals Announces Phase 2 Positive Clinical Results for EPI-743 in Rett Syndrome
Date:9/2/2014

n shown to correlate with disease severity and progression.  A recently published study, led by Claudio De Felice, MD, Professor of Neonatology and a co-investigator in the clinical trial, has documented the relationship between levels of oxidative stress markers in the brain and the disease in an in vivo disease model.

Rett syndrome affects one in every 10,000 to 15,000 live female births. The majority of cases result from a mutation in the MeCP2 gene that is carried on the X chromosome. Rett syndrome is typically diagnosed between six months and 18 months of age. Children then progress through the four stages of Rett syndrome, which are characterized by worsening neurological status and seizures. Death typically occurs prior to age 40. There is no cure for Rett syndrome and therapy is directed at management of disease symptoms.

EPI-743
EPI-743 is an orally bioavailable small molecule being developed by Edison Pharmaceuticals for the treatment of disorders of energy metabolism. Currently EPI-743 is in phase 2 clinical development.

Edison Pharmaceuticals
Edison Pharmaceuticals is focused on developing treatments for children and adults with orphan mitochondrial diseases.


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SOURCE Edison Pharmaceuticals, Inc.
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4. Edison Pharmaceuticals Announces Positive Results of EPI-743 Phase 2A Leigh Syndrome Clinical Trial
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