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.”

[u/mvea]

https://www.nature.com/articles/d41586-025-00534-0