MELAS and SRT501
Disease background and scientific rationale
MELAS syndrome (Mitochondrial myopathy, Encephalopathy, Lactic Acidosis, and Stroke-like episodes), an inherited disorder, is caused by a mutation in the DNA of mitochondria. MELAS mainly affects muscles and nerves, leading to reduced exercise capacity and eventually muscle failure. Patients with MELAS develop muscle disease, Type 2 Diabetes and lactic acid buildup. The disease is progressive and fatal and currently there is no known treatment.
We believe there are fewer than 20,000 MELAS patients in Europe and a smaller number in the United States.
Given the association of SIRT1 activators with increased function of mitochondria, we are currently testing SRT501 for the treatment of MELAS.
We expect data from this Phase 1b study during the first half of 2009.
To help you learn more about MELAS, we have provided the web links below:
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