Toddler with fatal genetic disease given world's most expensive drug - but it's too late to save sister
A 19-month-old girl has been successfully treated for a fatal genetic disease with the world's most expensive drug.
Teddi Shaw is the first person in the UK to receive Libmeldy, a treatment that corrects the underlying genetic fault that causes metachromatic leukodystrophy (MLD).
The gene therapy, which replaces a defective gene inside the body's cells, has a list price of £2.8m.
Even with a "significant confidential discount" by UK-based biotech company Orchard Therapeutics, NHS England says it is still the most expensive drug licensed in Europe.
But Teddi now shows no sign of MLD, a previously incurable disease that causes serious damage to the nervous system and organs.
Newly diagnosed patients have a life expectancy of just five to eight years.
Teddi's mum, Ally, said: "Teddi is doing absolutely brilliant. She is walking, running, a chatterbox, absolutely no signs so far of MLD.
"She is an absolute character and has everyone around her laughing all the time."
Teddi and her three-year-old sister Nala were both diagnosed with MLD in April last year.
Nala was unable to have treatment because of clinical guidance that it should start before the irreversible damage caused by the disease progresses too far.
Ally said: "Being told our first daughter Nala wasn't eligible for any treatment, would continue to lose all functions, and die extremely young was the most heartbreaking and hardest thing to come to terms with.
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