VIDEO: Disease progression proves great challenge in Friedreich’s ataxia management

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The biggest challenge right now in FA care is find something which stops progression. If we did that, then I think most people would do well with the level they are at now. If we were restored frataxin to normal in every cell in the body, you'd see probably an arrest of disease, although I won't stake my life of that, and you'll probably see improvement in some domains, which would be great. And people with FA are intelligent, they're well compensated. They've learned how to live with their disability. So if we just stopped it, it would be a very big deal.