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

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…
Source: Mayo Clinic Research News - Category: Research Source Type: news