Novartis discloses deaths of 2 children on spinal atrophy drug

Novartis, a Switzerland-based pharmaceutical company, confirmed a report of two children dying from liver complications six weeks after taking its gene therapy drug, Zolgensma, Bloomberg reported Aug. 11. 

The two deaths, which happened in Russia and Kazakhstan, occurred after a Zolgensma infusion and steroid doses intended to fight liver complications, according to Bloomberg

Zolgensma is used for spinal muscular atrophy, a rare condition that's present in 1 in every 10,000 people, according to the National Organization for Rare Disorders. Because this is the first report of deaths associated with the drug, Novartis will update the side effects label. 

"While this is clinically important safety information, it is not a new safety signal and we firmly believe in the overall favorable risk/benefit profile of Zolgensma, which to date has been used to treat more than 2,300 patients worldwide across clinical trials, managed access programs, and in the commercial setting," a Novartis spokesperson told Bloomberg.

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