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Girl with fatal MLD disease is cured with gene therapy
The Guardian
|February 15, 2023
A girl born with a rare and fatal genetic condition is expected to live a long and normal life after becoming the first person to be cured on the NHS with the help of a revolutionary gene therapy.
Teddi Shaw was diagnosed with metachromatic leukodystrophy (MLD), an inherited condition that causes catastrophic damage to the nervous system and organs. Those affected die young.
But the 19-month-old from Northumberland is now disease-free after being treated with the world's most expensive drug, Libmeldy. NHS England reached an agreement with its maker, Orchard Therapeutics, to offer it to patients at a significant discount from its list price of £2.8m.
"Teddi is doing absolutely brilliant," said her mother, Ally Shaw. "She is walking, running- absolutely no signs so far of MLD."
The chief executive of NHS England, Amanda Pritchard, said the breakthrough meant children such as Teddi could do the things that every child should be able to. Those born with the condition could now lead normal, healthy lives, she added.
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