by UA 19-month-old baby diagnosed with a fatal genetic disease was saved by gene therapy treatment by the UK’s National Health Service (NHS), which negotiated with the relevant drug company to reduce the tabulated amount to around three million pounds (about 3.3 million euros).
Little Teddi Shaw has become the first NHS patient to be treated for metachromatic leukodystrophy (MLD), a rare condition which destroys organs and the nervous systemwhich occurs when the body it cannot break down certain lipids because it does not have enough enzymes.
The life expectancy for people diagnosed with this disease before the age of 30 months is between five and eight yearswhich means that Teddi would have died in infancy if the treatment – which removes the stem cells and replaces the defective gene before re-injecting the treated cells – was not available. If the condition begins after the age of 30 months and before the age of six, there is a life expectancy of between 10 and 20 years.
The treatment, known by the brand name Libmeldy, was the most expensive drug in the world when the NHS negotiated a confidential discount deal with makers Orchard Therapeutics a year ago.
It remains the most expensive drug licensed in Europe to be dispensed in the UK exclusively in a dedicated unit at the Royal Manchester Children’s Hospital. It also applies to four other locations in Geria Epirus.
“This is a moment of hope for parents and children with terrible illnesses. Now they can get the right treatment in the British National Health System,” said Amanda Pritchard, director of the service.
The Shaw family, however, did not leave without a bitter aftertaste. While Teddi could access the treatment, his older sister Nala, three, who was also diagnosed with the same disease, is not eligible as she is at an advanced stage of the disease.
“We are extremely privileged that Teddi is the first child to receive this on the NHS and are grateful that she has the chance to live a long and hopefully normal life. Without this treatment, we would have faced having our two daughters taken from us.. We can only hope that one day a treatment will be available for all stages of MLD, and we strongly believe that it should be added to the newborn screening test to prevent more families from going through this headache,” reported British newspaper The Independent. .
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