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Two children have died from acute liver failure after being administered Zolgensma, a pricey gene therapy sold by Novartis to treat a rare disease.

The company this week began notifying physicians and payers about the deaths, which occurred over the past few months in Russia and Kazakhstan. The children passed away approximately five to six weeks after the one-time therapy was infused and between one and 10 days after corticosteroid tapering occurred, which is done to ensure proper liver function.


Although acute liver failure is noted in the product labeling information, a Novartis spokesperson acknowledged these episodes marked the first fatal cases attributed to the side effect. There were no deaths tied to acute liver failure in clinical trials for the medicine, which is used to treat spinal muscular atrophy, a genetic disorder that causes muscle wasting and weakness.

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