NHS to offer 'world's most expensive' £1.8m-a-dose drug to cure baby spinal ... trends now
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Babies born with a genetic disease that leads to a life expectancy of just two years without intervention will now be cured on the NHS with a new £1,8million drug.
Infants with severe spinal muscular atrophy (SMA), which causes muscle weakness and leading to paralysis and breathing problems, will now be able to take the gene therapy drug Zolgensma.
Treatment watchdog The National Institute for Health and Care Excellence (Nice) has approved the use of onasemnogene abeparvovec, known more commonly by its brand name.
Around 65 babies born every year in England have spinal muscular atrophy, which is the leading genetic cause of death in children.
The disease causes a defect in a gene called SMN1, which makes essential protein and thus enables spinal nerves in the spinal cord to control the movement of muscles.
Then five-month old Arthur Morgan with his father Reece Morgan in June 2021 after he became the first patient in England to undergo gene therapy using Zolgensma
The most sever form of the condition, Type 1 SMA, can develop in infants less than six months old - with a 90 per cent chance of dying before the age of two if untreated, The Times reports.
But Zolgensma has been proven in studies to
