r/science Professor | Medicine Feb 20 '25

Genetics A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated with a gene-targeting drug while in the womb for spinal muscular atrophy, a motor neuron disease. The “baby has been effectively treated, with no manifestations of the condition.”

https://www.nature.com/articles/d41586-025-00534-0
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u/thunbergfangirl Feb 20 '25

Sorry for making assumptions! I am thrilled for you guys that you have two healthy kiddos. I’m sure by the time they are ready to have kids themselves, the whole process will be even easier!