Grand Rapids, Mich.-based Helen DeVos Children's Hospital provided a $2.1 million gene therapy drug for spinal muscular atrophy to a one-year-old girl in November, reports Fox 47 News.
Five things to know:
1. SMA is a rare genetic disease that leads to progressive muscle weakness and, in some cases, paralysis.
2. This was the second patient to receive the treatment at Devos Children's.
3. AveXis developed the drug, which is called Zolgensma. AveXis was acquired by Novartis for $8.7 billion in 2018.
4. The FDA approved Zolgensma in May. It is the most expensive drug in the world, according to The Washington Post.
5. AveXis told Fox 47 News that the cost represents long-term value for SMA patients and is approximately 50 percent "less than the current 10-year cost of chronic SMA therapy."
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