Words by Abi Scaife
A young girl with a fatal genetic condition has been cured.
Incredible! Tell me more.
Teddi Shaw, aged 19 months, was born with metachromatic leukodystrophy (MLD). This is a hereditary disease that targets nerves and organs and sadly means that those affected die young.
However, in Teddi’s case, an incredible new drug means she will likely go on to live a long, normal and healthy life.
How does it work?
A brand new gene therapy drug was given to her on the NHS. The retail price of the drug is around £3 million, however, the NHS was given an ‘undisclosed’ discount.
The drug works by inserting functional copies of a faulty gene back into the patient’s stem cells. The stem cells are taken from the patient's body, edited, and then reintroduced with all the new information.
It is hoped that this new treatment will go on to help young children across the UK go on to live long, healthy lives.