Arthur Morgan received the one-off gene therapy at Evelina London Children’s Hospital on 25 May.
‘This treatment will bring hope to families affected by SMA who have fought so courageously against it’
There were no treatment options available for children with spinal muscular atrophy (SMA) - which is the leading genetic cause of death for children - until two years ago.
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.
However, studies found that a single treatment with US gene therapy Zolgensma has helped babies with SMA to sit, crawl and walk and also prevented them from having to be put on a ventilator.
Zolgensma has been labelled the most expensive drug in the world, with a list price of £1.79 million per dose.
It was made available on the NHS after the health service made a deal with manufacturers Novartis Gene Therapies in March of this year.
Arthur, who was diagnosed with SMA earlier this month, underwent the gene therapy infusion last week.
His father, Reece Morgan, 31, 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.”
Four specialist NHS centres have now been commissioned across the country to administer the treatment, including Evelina London Children’s Hospital, where Arthur was treated.
The other sites are located at 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.”