We use your sign-up to provide content in ways you’ve consented to and to improve our understanding of you. This may include adverts from us and 3rd parties based on our understanding. You can unsubscribe at any time. More info
Toddler Teddi Shaw is the first UK child to receive a gene therapy treatment costing millions on the NHS for a rare deadly genetic condition.
The 20-month-old inherited life-limiting condition metachromatic leukodystrophy (MLD). It affects one in 40,000 babies and causes loss of nervous system and brain function, leading to a life expectancy of between five and eight years.
Revolutionary treatment Libmeldy, with a list price of £2.8million, is a one-off gene therapy and was the most expensive drug in the world when NHS England started negotiating a significant discount.
It works by removing the child’s stem cells and replacing the faulty gene that causes MLD before re-injecting the treated cells.
Now Teddi has become the first UK child to have Libmeldy on the NHS. She started treatment last June and is now happy and healthy, recovering back home in Northumberland.
Sadly, Teddi’s sister Nala, three, also has MLD but was medically assessed as being unsuitable due to her age to try the therapy.
The treatment is available on the NHS as a specialist service and in England is being delivered at Royal Manchester Children’s Hospital – one of just five sites in Europe administering the treatment.
Mum Ally Shaw, 32 – married to the girls’ father Jake, 29 – said: “Teddi is doing absolutely brilliantly! She is walking, running, a chatterbox, absolutely no signs so far of MLD.
“In April last year, our world was turned upside down when not one, but both of our daughters were diagnosed with MLD.
“Being told our first daughter, Nala, wasn’t eligible for any treatment and would die extremely young was heartbreaking. However, among the pain was hope for Teddi.
“We are privileged Teddi is the first child to receive this on the NHS. Without this, we would be facing both our children being taken away.”
Libmeldy, made by UK firm Orchard Therapeutics, is a one-time treatment that corrects the underlying cause of MLD.
NHS chief executive Amanda Pritchard said: “Thanks to advancements in gene therapies, and the commercial ability of the NHS to strike deals for cutting-edge drugs, children born with this condition now have the opportunity to lead normal, healthy lives.”
Professor Rob Wynn, of Royal Manchester Children’s Hospital, said: “It is wonderful to be involved in this breakthrough moment.”
Source: Read Full Article