A five-month-old baby suffering from a spinal disorder has become the first NHS patient in England to be treated with a potentially life-saving drug that can prolong life.
Arthur Morgan was diagnosed with spinal muscular atrophy (SMA) earlier this month.
He received Zolgensma, a one-time American gene therapy, on May 25 at Evelina London Children’s Hospital.
The treatment has been called the world’s most expensive drug – with a list price of £ 1.79m per dose.
In March it was made available on the NHS following an agreement with healthcare Novartis Jean Therapies.
Baby Arthur, who was born six weeks early in December, underwent a gene therapy infusion last week after being diagnosed with SMA less than three weeks ago.
Until two years ago, there were no treatment options available for children with the condition.
His father, Rees Morgan, who works as a self-employed plasterer, said: “When we learned that Arthur would be getting treatment, and would be the first patient, I just broke down.
“It was a whirlwind couple of weeks, filled with a lot of anxiety and adjustment, as we learned about his condition and what it could mean for him and our family.”
The 31-year-old said: “We still don’t know what will happen in the future, but it gives Arthur the best chance of giving him the best possible future.”
Children born with type 1 SMA – the most common form of the condition – experience progressive muscle weakness, loss of movement, difficulty breathing, and have a life expectancy of only two years.
But studies have found that a single zolgensma treatment can help infants sit, crawl, and walk, and prevent them from being placed on ventilators.
Four specialist NHS centers across England can now treat, including Evelina London Children’s Hospital, where Arthur was treated.
Other sites are Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust and University Hospital Bristol and Weston NHS Foundation Trust.
Dr Elizabeth Wrige, a pediatric neurologist at Evelina London Children’s Hospital, said: “This treatment will bring hope to families affected by SMA who have fought so courageously against it.”
Sir Simon Stevens, chief executive of the NHS, also said: “It is great news that this revolutionary treatment is now available on the NHS for babies and children like Arthur.
“The NHS Long Term Plan is committed to providing state-of-the-art treatment for patients at a reasonable cost to taxpayers.
“Zolgensma is the latest example of the life-changing therapy that the NHS is now routinely using to transform the lives of patients and their families.”
Health Secretary Matt Hancock said: “I am very pleased that young Arthur can access this potentially life-changing treatment on the NHS. I hope it gives new hope to his family and many others that the lives of more children Can be replaced. “
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