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.
