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Rare Genetic Disorder Treated in Womb For the First Time
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. Nature: The child's mother took the gene-targeting drug during late pregnancy, and the child continues to take it. The "baby has b...
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 "baby has been effectively treated, with no manifestations of the condition," says Michelle Farrar, a paediatric neurologist at UNSW Sydney in Australia. The child was conceived with a genetic condition known as spinal muscular atrophy, which affects motor neurons that control movement, and leads to progressive muscle weakening.
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