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Gene therapy may have its first blockbuster

Novartis will sell the world’s most expensive drug, a treatment called Zolgensma to treat spinal muscular atrophy.
by Antonio Regalado

A newborn. A fatal diagnosis. And soon, a one-time gene replacement cure in the first weeks of life.

The cost? You don’t want to know.

Gene therapy is about to achieve a milestone. As soon as tomorrow, drug giant Novartis expects to win approval to launch what it says will be the first “blockbuster” gene-replacement treatment. A blockbuster is any drug with more than $1 billion in sales each year.


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