NHS is offering £1.8million of medicine to help cure babies with spinal conditions
Babies born with a genetic condition resulting in a life expectancy of just two years without intervention are now being cured in the NHS with a new £1.8million drug.
Infants with severe spinal muscular atrophy (SMA), which causes muscle weakness and leads to paralysis and breathing problems, can now take the gene therapy drug Zolgensma.
Treatment Guardian The National Institute for Health and Care Excellence (Nice) has approved the use of onasemnogene abeparvovec, better known by its brand name.
Around 65 babies born in England each year suffer from spinal muscular atrophy, the leading genetic cause of death in children.
The disease causes a defect in a gene called SMN1, which makes an essential protein that allows the spinal nerves in the spinal cord to control muscle movement.
Arthur Morgan, then five months old, with his father Reece Morgan in June 2021 after becoming the first patient in England to undergo Zolgensma gene therapy
The most severe form of the disease, type 1 SMA, can develop in infants as young as six months – with a 90 percent chance of dying before the age of two if left untreated, The Times reports.
But Zolgensma has shown in studies that it helps sufferers sit, crawl and walk – things they would never normally be able to do – and also saves them from being put on a ventilator.
The single infusion, administered in just one hour, uses a harmless virus to deliver a healthy version of the SMN1 gene.
It enters nerve cells to replace the defective gene and restores normal function, allowing the baby to live a healthy life with minimal symptoms.
It comes after year-old Edward Willis-Hall was given the drug and subsequently took his first assisted steps in January earlier this year.
His mother Megan Willis, 30, spoke of her joy after her son reached milestones she never thought possible.
He can roll over, sit up, stand for five minutes in gaiters that support his legs, and take steps in a harness.
She told the BBC: “We are so proud of Edward. He’s doing incredibly well. He far exceeds our expectations. We are so incredibly happy.’
Ms Willis, who was told her son suffered from spinal muscular atrophy by the age of seven weeks, set a goal for him to sit unaided. She said: “All I ever wanted was for him to be able to sit, and I knew then he would have an amazing life.”
Nice said buying the drug was a “low-cost” use of resources.
Only babies with a family history of SMA are currently tested.
The single infusion, administered in just one hour, uses a harmless virus to deliver a healthy version of the SMN1 gene
But activists are urging every five-day-old infant to have screenings that diagnose nine diseases.
Laurent Servais, Professor of Pediatric Neuromuscular Diseases at the University of Oxford, said: “I’ve seen too many families devastated by this disease, but we now have treatment options.
“However, it is still too late to wait for symptoms to appear. Every day we delay in finding and treating these infants, we could be responsible for a child spending its life in a wheelchair.
“There is 100 per cent consensus among UK experts, among world experts and among patients: we must screen to save lives, we must screen to preserve quality of life and we must screen to save money.”
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https://www.soundhealthandlastingwealth.com/health-news/nhs-to-offer-1-8million-drug-to-cure-babies-of-spinal-disease/ NHS is offering £1.8million of medicine to help cure babies with spinal conditions