A 19-month-old baby girl called Teddi has become the first child in the UK to receive a life-saving gene therapy treatment for the fatal disorder, metachromatic leukodystrophy (MLD).
The revolutionary gene therapy, known by its brand name Libmeldy®, has a list price of £2.8 million and was the most expensive drug in the world when NHS England negotiated a significant confidential discount last year to make the treatment available to NHS patients – it remains the most expensive drug licensed in Europe.
The genetic disease causes severe damage to the affected child’s nervous system and organs, resulting in a life expectancy of between just five and eight years…
