A baby with a rare inherited disorder became the first child in the U.K. to receive a new gene therapy for the condition.
A 19-month-old girl named Teddi recently became the first child in the U.K. outside a clinical trial to receive a new gene therapy for metachromatic leukodystrophy , a fatal genetic disorder, the National Health Service announced .
The genetic disorder MLD disrupts cells' ability to break down sulfatides, a fatty material used to insulate the wiring that runs through the white matter of the brain and much of the nervous system beyond the brain. Sulfatide buildup destroys brain and nerve cells, resulting in cognitive problems, a loss of motor control and sensation, seizures, paralysis and blindness, according to the Genetic and Rare Diseases Information Center . Eventually, the disorder leads to death.
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