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Infant with Spinal Muscular Atrophy Saved by $2 million Drug

Published September 30, 2019
Category: Medication

Doctors started her on a drug called Spinraza, which has shown promise in babies with SMA since it was approved by the FDA in 2016. It requires an injection into their tiny bodies every four months for the rest of their lives.Not long after Kaeli’s initial treatment on Spinraza, a newly approved gene therapy drug called Zolgensma hit the market. It would essentially give Kaeli’s body the missing gene, and it would only require one dose. However, the drug came with a price tag of $2.1 million.

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Last change: September 30, 2019


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