Toddler denied $2.1m gene therapy will now get it for free

Jonathan Saltzman — Boston Globe

A 2 1/2-year-old girl who was deemed ineligible for the world’s most expensive drug will get the $2.1 million medicine to treat her rare genetic disease after all.

AveXis, a subsidiary of the Swiss drug giant Novartis, has agreed to provide Zolgensma, an intravenous gene therapy that requires only one dosage, free to Natalia Boidi through a compassionate use program. She has spinal muscular atrophy, which kills more infants than any other inherited disorder.

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