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Mayo Clinic discovery leads to life-changing treatment for young girl with ultra-rare disease

March 3, 2023
Rare Disease Day on Feb. 28 raises awareness of the 30 million people in the U.S. who have a rare disease. Months after young Maggie Carmichael started taking an experimental drug for her ultra-rare genetic disease, she was able to trade in her wheelchair for a walker. The 9-year-old Mayo Clinic patient even took a few of her first-ever steps on her own. She also became better at feeding herself, her speech improved and she…

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