Little girl's life saved in Manchester by new gene therapy treatment in UK first
15.02.2023 - 09:37
/ manchestereveningnews.co.uk
A little girl has been become the first patient in the UK to get life-saving gene therapy treatment for a fatal disorder on the NHS - in Manchester. In children, genetic disease metachromatic leukodystrophy - MLD for short - causes severe damage to the nervous system and organs, resulting in a life expectancy of between just five and eight years.
The most common form of MLD usually develops in babies younger than 30 months and can lead to loss of sight, speech and hearing, as well as movement difficulty, brain impairment and seizures, said the NHS. The genetic disorder prevents the development of a crucial enzyme that leads to a build-up of fats that destroy protective layers around a child's nerves.
Teddi Shaw, now aged 19 months, and her older sister Nala, three, were both diagnosed with MLD in April last year. Nala, said the NHS, is tragically not eligible for the treatment as clinical guidance requires the gene treatment to be administered before the irreversible damage caused by the disease progresses too far.
She's said to be too far advanced to benefit, although those whose MLD starts between 30 months and six years of age have a life expectancy of 10 to 20 years more.
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But NHS bosses have revealed today Teddi was the first person in the UK to receive the treatment outside of a clinical trial and is now a happy and healthy toddler, showing no signs of the devastating disease she was born with.
The gene therapy - known by its brand name Libmeldy- works by removing a child's stem cells and replacing the faulty gene that causes MLD before re-injecting the treated cells into the patient.
Teddi's treatment began when she was 12 months old with the removal of stem cells at the end of last June, which were