Novartis drug causes two children to die of liver failure from gene therapy drug


Novartis Pharmaceuticals is reporting that two children have died after taking Zolgensma, a gene therapy drug designed to treat a rare condition called spinal muscular atrophy.

In an email statement, Novartis says the children died from acute liver failure about six weeks after receiving an infusion of Zolgensma, and one to 10 days after tapered doses of a steroid meant to combat liver complications.

The deaths occurred in Russia and Kazakhstan. 

The FDA approved Zolgensma in the U.S. in 2019 and is estimated to cost more than $2 million per patient.

Read more here.


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3 thoughts on “Novartis drug causes two children to die of liver failure from gene therapy drug”

  1. Seeing how over 40% of the FDA’s annual budget is received by drug makers through user fees paid, thanks to Corrupt Congress passing legislation to do so….we all ply Russian Roulette with drugs and medical devices. A woman I know left the FDA because she was being pressured to approve unsafe and unproven drugs and devices. Sleep well, folks!

    1. I’m not sure I understand your post; the FDA gets funding by big pharma? I thought it was funded just by our taxpayer dollars. What are ‘user fees’, and how does Congress play into this? Sorry for the questions~I don’t know that much about this subject.

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