A five-month-old baby has become the first patient in England treated with a potentially life-saving drug on the NHS that can prolong the lives of children with spinal muscular atrophy.
Arthur Morgan, who was diagnosed with the condition earlier this month, received the one-off gene therapy at Evelina London Children’s Hospital on May 25.
But babies could potentially have the ability to sit, crawl and walk after being treated with US gene therapy Zolgensma, which has been called the most expensive drug in the world.
Zolgensma, which has a list price of £1.79 million per dose, was made available on the NHS after the health service struck a deal with manufacturers Novartis Gene Therapies in March.
His father Reece Morgan, 31, who works as a self-employed plasterer, said: “When we found out that Arthur would get the treatment, and be the first patient, I just broke down.
“It had been such a whirlwind few weeks, filled with lots of anxiety and adjustment, as we learnt about his condition and what it might mean for him and our family.
“We still don’t know what the future will hold, but this gives Arthur the best possible chance to give him the best possible future.”
Babies born with Type 1 SMA, which is the most common form of the condition, experience progressive muscle weakness, loss of movement, difficulty breathing, and have a life expectancy of just two years.
Studies found that a single treatment with Zolgensma has helped babies with SMA to sit, crawl and walk, and also prevented them from having to be put on a ventilator.
The other sites are Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust and University Hospitals Bristol and Weston NHS Foundation Trust.
Dr Elizabeth Wraige, consultant paediatric 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.”
NHS chief executive Sir Simon Stevens added: “It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur on the NHS.
“The NHS Long Term Plan committed to securing cutting edge treatments for patients at a price that is fair to taxpayers.
“Zolgensma is the latest example of the life-changing therapies that the NHS is now routinely using to transform the lives of patients and their families.”
Health Secretary Matt Hancock said: “I am so glad young Arthur can access this potentially life-changing treatment on the NHS. I hope it grants his family and many others renewed hope that more children’s lives can be transformed.”