At $2.1m a dose, this is the world’s costliest treatment for a disease

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The price will be $2.1 million, believed to be highest ever set for a one-time treatment. The therapy, to be sold as Zolgensma, alters the underlying genetic cause of spinal muscular atrophy and may permanently stop the disease. It is among the first of gene therapies that promise a cure for deadly inherited conditions.



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