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Zolgensma, a new drug approved by the FDA Friday, costs more than $2.1 million. It's made by AveXis, a drugmaker owned by pharmaceutical giant Novartis. Bloomberg/Bloomberg via Getty Images hide caption

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Bloomberg/Bloomberg via Getty Images

At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever

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Seven-year-old Carson Miller (left), and his brother, 5-year-old Chase Miller (right), both have a degenerative brain disease called MEPAN syndrome. There are only 13 people in the world who have it. Courtesy of Andrew Ross-Perry hide caption

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Courtesy of Andrew Ross-Perry

Medical Detectives: The Last Hope For Families Coping With Rare Diseases

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The Food and Drug Administration has seen a sharp increase in applications for drug to treat rare diseases. An oversight report found problems with how agency is handling them. Al Drago/CQ Roll Call/Getty Images hide caption

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Al Drago/CQ Roll Call/Getty Images

Connor Webb and his mother, Kim Webb, stand outside of their home in Huntington Beach, Calif. Connor was treated for a rare cancer at 16. He's well now but his mother is fighting for new cures in case the cancer comes back. Alex Welsh for NPR hide caption

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Alex Welsh for NPR