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The Making of Medicine

Friendships Made in the Fight Against Friedreich’s Ataxia

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Transcript: This is a featured video transcript

I wanted to share this great video with you. It’s about a genetic disease called Friedreich’s ataxia, and the releationships our Zhen Yan, PhD, has made while researching the condition. It’s a wonderful story of how science can bring people together, and the friendships that can spring from that.

Professor Yan’s latest findings suggest that well-timed exercise early in life could slow the progression of the disease, which robs patients of the ability to walk.

In a lab model of the disease, mice lose the ability to run, develop blood sugar problems and show signs of moderate heart problems by the time they’re 6 months old. But mice that started voluntary long-distance running at 2 months completely avoided those problems, Yan found.

Yan’s work is in mice, but he’s hoping a clinical trial will see if the findings hold true in people.

“When dealing with a genetic disease, we often hope that gene therapy is advanced to a point with great precision and efficiency that we can replace the defect gene in the whole genome and in all the affected cells in the body, but the reality is that we are not there yet,” he told me. “This could fundamentally change for the good the life of Friedreich’s ataxia patients.”

Yan, a top exercise researcher, has published several pieces of fascinating research lately, including his finding that exercise could help prevent a deadly complication of COVID-19.

Finally, I should mention that the video at top is by my wonderful colleague Harry Moxley. Thanks, Harry.


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