Two Children Died With Sma: "Liver Dysfunction After Gene Therapy"

Serious follow after therapy Anti-Sma gene (spinal muscular atrophy) Zolgensma by Novartis. In the case of liver dysfunction, they are dead two children in Russia and Kazakhstan. This was announced by a Swiss pharmaceutical company, which said: “After two deaths in a short period of time and in agreement with the health authorities – according to the position of the Basel giant – we will update the warnings “on treatment” and pay attention to the fact that fatal ones Cases of acute hepatic dysfunction have been registered.

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Both patients received steroids after gene therapy. According to Novartis, these are the first deaths from liver dysfunction after treatment. But the company reaffirms its confidence in the drug: “We firmly believe in the cost-benefit ratio of Zolgensma, which has been used in over 2,300 patients worldwide,” emphasizes the multinational corporation.

Possible hepatic side effects were already known and were specified in the product description. Zolgensma is approved for the treatment of SMA: a illness rare inherited disorder that leads to death in its most severe form around the age of 2 years. The $2 million drug is known to be the most expensive single therapy in the world. Zolgensma was approved in the US in 2019 and in the EU in 2020.