Typically diagnosed in early childhood or adolescence due to increased clumsiness, Friedreich’s ataxia (FA) is a progressive neurodegenerative disease that worsens over time. Eventually, most patients will lose motor function and become confined to a wheelchair. Many will also suffer from heart failure.
As it stands, there is no cure or effective treatments to slow or prevent the progression of FA. However, scientists at the Scripps Research Institute (TSRI) are working on several therapeutic options that have shown promise in treating Friedreich’s ataxia. By studying the molecular basis of the disease, researchers discovered a small molecule that could reactivate the functionality of the gene that’s responsible for the development of the disease.
Thanks to the support of a few different pharmaceutical companies, TSRI has been able to take these learnings, develop drugs and begin studying their effects on animals. They’ve now also completed a phase 1 clinical trial and are hoping to make some adjustments to the treatment and continue with additional clinical trials in the near future.
Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.