First baby receives life-saving gene therapy on NHS
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 a fatal disorder. Doctors caring for Teddi and her sister suspected the children were affected by metachromatic leukodystrophy (MLD). 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.
The diagnosis was provided by by a rapid gene test delivered by the NHS South West Genomic Laboratory, part of the national NHS Genomic Medicine Service. It resulted in Teddi getting treatment via a pioneering stem cell therapy means she is now a happy, healthy toddler, with no signs of the devastating disease she was born with.
The diagnosis meant Teddi was able to receive a revolutionary gene therapy, known by its brand name Libmeldy®. It 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.
https://sites.exeter.ac.uk/exeterbrc/2023/03/06/first-baby-receives-life-saving-gene-therapy-on-nhs/
ارسال به دوستان