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Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy

Published December 13, 2019
Category: Innovation
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Earlier this year, the US Food and Drug Administration approved the most expensive drug ever to hit the market, a gene therapy for spinal muscular atrophy. The genetic correction is currently used to treat affected newborns, but as symptoms for some types of SMA may appear before birth, an earlier treatment would be potentially more effective. 

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Last change: December 13, 2019

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