Rare genetic disorder treated in womb for the first time

The Independent

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This rare genetic disorder was just treated in the womb for the first time ever

Without treatment, those with the more severe form of illness may die before their second birthday

Greek Reporter

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Rare Genetic Disease Treated for the First Time in the Womb

A baby in the womb. Credits: rchristie/Ryan, CC BY-NC 2.0, via Wikimedia Commons. A two-and-a-half-year-old girl was successfully treated for a rare genetic motor neuron disease. In a groundbreaking study, the patient began receiving the treatment while still in the womb. The mother took a drug that directly targeted the disease, spinal muscular atrophy, and the girl continues to take it.  Spinal muscular atrophy is a genetic disorder that targe…

Nature

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Rare genetic disorder treated in womb for the first time

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

New Atlas

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Infant's genetic muscle disorder improved by treating pregnant mother

Spinal muscular atrophy is a debilitating genetic condition that’s usually fatal by a few years of age. But an intriguing case study might demonstrate a simple new treatment, with a child showing no signs at all two and a half years after birth.

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In the silence of a Sydney laboratory, a couple of parents and a scientific team answered a question that changes the history of medicine: “What if you could cure a person before they are even born?”. That question, born from the pain of a previous loss caused by spinal muscular atrophy (SMA), a devastating genetic disorder that affects motor neurons causing progressive muscle paralysis, kicked off incredible fetal therapy. Today, two and a half…

slashdot.org

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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 been effectively treated, with no manifestations of the condition," says Michelle Farrar, a paediatric neurologist at UNSW Sydney in Australia. The re…