04/16/2026
From Breakthroughs to Hope
Duke scientists are shaping the future of genetic medicine, developing powerful new ways to treat rare diseases like Pompe disease and glycogen storage disorders. By advancing gene therapy and genome editing, this research moves us closer to longer‑lasting, life‑changing treatments for patients and families. 💙🔬
Learn more:
https://medschool.duke.edu/news/pioneering-next-generation-genetic-therapies
In 2006, Duke University School of Medicine researchers, led by YT Chen, MD, PhD, professor emeritus of pediatrics, secured the Food and Drug Administration’s approval for a breakthrough enzyme replacement therapy called Myozyme to treat Pompe disease. Pompe disease is a glycogen storage disease, ...