Research Update: Treating Friedreich’s Ataxia
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.
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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.
MORE: Five types of treatment for Friedreich’s ataxia
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