New Gene Therapies May Soon Treat Dozens of Rare Diseases, but Million-dollar Price Tags Will Put Them Out of Reach For Many
Zolgensma – 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 costs US$2.1 million.
Read more here: New Gene Therapies
Kevin Doxzen, Hoffmann Postdoctoral Fellow, Arizona State University