Did you know?
A 19-month-old girl named Teddi recently became the first child in the UK outside a clinical trial to receive a new gene therapy for metachromatic leukodystrophy (MLD), a fatal genetic disorder. Roughly six months out from treatment, Teddi is a happy and healthy toddler showing no signs of the devastating disease she was born with. The genetic disorder MLD disrupts cells’ ability to break down sulfatides, 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. Eventually, the disorder leads to death.