Rare genetic disorder treated in womb for the first time

February 20, 2025

3D rendering of a DNA molecule

(Nature) – The child, who is now almost three years old, shows no signs of the often fatal motor neuron disease.

A two-and-a-half-year-old girl shows no signs of a rare genetic disorder, after becoming the first person to be treated for the motor-neuron condition while in the womb. The child’s mother took the gene-targeting drug during late pregnancy, and the child continues to take it.

The “baby has been effectively treated, with no manifestations of the condition,” says Michelle Farrar, a paediatric neurologist at UNSW Sydney in Australia. The results were published in the New England Journal of Medicine yesterday. (Read More)