Minnesota toddler with rare genetic disorder who received world's most expensive drug is now walking

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Minnesota toddler with rare genetic disorder who received world's most expensive drug is now walking
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The treatment, known as Zolgensma, has a price tag of $2.1 million per patient.

Before Smith's second birthday—the cut-off point to receive the new drug treatment—the toddler was unable to talk or even crawl around, being forced to scoot on the floor if she wanted to move.The gene therapy—which was developed by Novartis and recently approved by the U.S. Food and Drug Administration—is known as Zolgensma. It has proven controversial due to the eye-watering price tag of $2.1 million per patient, which makes it the world's most expensive drug.

Smith received the drug treatment—which is administered in a single dose—just a day before she turned two, becoming the first patient in Minnesota to be treated with the drug.

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