From Sept. 27 to Sept. 30, the second International Ataxia Research Conference is taking place in Pisa, Italy. Like the inaugural conference in 2015, the meeting will bring together patients, doctors, patient advocacy groups, researchers, pharmaceutical companies, regulators and others interested in the different forms of ataxia (including Friedreich’s).
The conference is organized by GoFAR, FARA, and Ataxia UK — three organizations that are dedicated to ataxia research. The hope is to advance “basic, translational and clinical science for ataxias.”
The four-day meeting will cover a variety of topics, from diagnosis to gene therapy. The event is coming at the perfect time, as ataxia research has made several promising developments in recent years.
Here are three topics that are sure to be a highlight of the conference:
1. Using gene therapy to treat ataxias.
One of the most exciting developments in ataxia research has been the recent discoveries in gene therapy. Jane Larkindale, FARA’s vice president of research and development, told Friedreich’s Ataxia News that, “Gene therapy has just made leaps and bounds in the last couple of years. There’s going to be a lot of discussion about that because it’s a big, exciting new area.” This will include talking about CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats.
2. Friedreich’s ataxia clinical trial reviews.
According to conference organizers, 19 presentations will cover new treatments. One of the presentations will review the results of the MOXIe clinical trial assessing Reata’s omaveloxolone therapy, which has shown promising results in treating Friedreich’s ataxia.
3. How crossover drugs can help ataxia patients.
It’s not just new treatments that will be a focus of the conference — many companies involved in the conference have drugs on the market for other diseases that may also benefit FA patients. Some of these drugs have been used to treat other neurological diseases like muscular dystrophy and Parkinson’s disease.
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.
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