Teddi Shaw, from Northumberland, first recipient on health service of Libmeldy, world’s most expensive drug
A girl born with a rare and deadly genetic condition is expected to live a long and normal life after becoming the first person to be cured on theTeddi Shaw was diagnosed with metachromatic leukodystrophy , an inherited condition that causes catastrophic damage to the nervous system and organs. Those affected usually die young.is now disease-free after being treated with the world’s most expensive drug, Libmeldy.
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, “like going to school and playing with friends”. People born with the deadly condition could now lead normal, healthy lives, she said. Libmeldy corrects the genetic cause of MLD by inserting functional copies of a faulty gene into the patient’s stem cells. The stem cells come from their own bone marrow or blood and are fed back into the body with the new genetic information.
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