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New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many

By Kevin Doxzen, Hoffmann Postdoctoral Fellow, Arizona State University

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…

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