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Drug maker Novartis launches lottery of 100 free doses of $2.1million gene therapy for children under 2 with leading genetic cause of infant death

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“SMA is still the biggest killer of children under two. Imagine parents putting a child in a draw every two weeks to see if their life can be saved. It will have a huge emotional impact,” she said. “As a mother of a child with SMA, I think it could have been done much better.”

The drug, Zolgensma, was developed by AveXis, which is owned by Novartis, one of the wealthiest drug companies in the world. It is designed to add a functional copy of a gene that is missing in babies born with SMA. It is not a cure.


Huge debate about this drug in the SMA community over the price and complete unknown of long term effectiveness, especially given the high cost of the alternative therapies. 

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