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.
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