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STAT Plus: Some insurers are pushing back on the high price for a Novartis gene therapy

Barely a month after Novartis (NVS) won U.S. regulatory approval for its gene therapy — the most expensive in the world — nearly a dozen insurers are taking steps to restrict coverage, a trend that is concerning for gene therapies more broadly, according to a Wall Street analyst.

At issue is Zolgensma, a gene therapy product for infants with spinal muscular atrophy, or SMA, an inherited disease that causes progressive loss of muscle function. The most severe form of SMA causes infants to die or need permanent breathing support by the age of 2. The  “one-and-done” treatment is considered a miracle cure by some, but the $2.125 million price tag is controversial.

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