New gene therapies may soon treat dozens of rare diseases, but million-dollar price tags will put them out of reach for many
September 3, 2021
Gene therapy uses our genomic makeup to treat or prevent disease. ktsimape/iStock via Getty Images 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 .
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