New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many
- Written by Kevin Doxzen, Hoffmann Postdoctoral Fellow, Arizona State University
Gene therapy uses our genomic makeup to treat or prevent disease. ktsimape/iStock via Getty ImagesZolgensma – which treats spinal muscular atrophy, a rare genetic disease that damages nerve cells, leading to muscle decay – is currently the most expensive drug in the world. A one-time treatment of the life-saving drug for a young child c...