JOT101

• Pre-clinical and development work has been in-licensed from Murdoch Childrens Research Institute, a renowned Australian research and treatment facility. 
• Studies performed with adult patients, with potential to expand into pediatrics. 
• Previously conducted clinical studies have shown efficacy in key, measurable endpoints.
• Phase II clinical trials have qualified for subsidized funding from the Australian government

What is Friedreich's Ataxia?

Friedreich's Ataxia (FA) is a rare inherited disease that causes damage to the nervous system as well as mobility dysfunctions. FA usually begins in childhood and leads to impaired muscle coordination (ataxia) which worsens over time. It is caused by a defect (mutation) in a gene labeled FXN. Friedreich's ataxia is recessive, meaning it only occurs in someone who inherits two defective copies of the gene, one from each parent. Although rare, FA in the most common form of hereditary ataxia, affecting about 1 in 50,000 people in the United States. As of 2015 there are still no approved treatments available.