Five-month-old baby becomes first NHS patient treated with drug for spinal muscular atrophy in UK

ITV News Correspondent Sejal Karia on Arthur's breakthrough moment

A five-month-old baby with a spinal disorder has become the first patient in England treated with a potentially life-saving drug on the NHS that can prolong life.

Arthur Morgan received the one-off gene therapy at Evelina London Children’s Hospital on May 25, after being diagnosed with a spinal condition in early May.

Baby Arthur is recovering well after being treated for a spinal condition with the most expensive drug in the world

Until two years ago, there were no treatment options available for children with spinal muscular atrophy (SMA), which is the leading genetic cause of death for children.

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.

Dad Reece Morgan describes how he noticed baby Arthur started to slow down with his movements after two months and how finding out about the gene therapy gave him hope

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.

It is not known what price the NHS and manufacturers agreed on.

Baby Arthur, who was born six weeks premature in December 22, underwent the gene therapy infusion last week after being diagnosed with SMA less than three weeks earlier.

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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.

Five-month old Arthur Morgan. Credit: PA

“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.”

He described how he noticed Arthur started to slow down with his movements after two months of being born. He took his son to the hospital after a GP said "age would correct itself".

Dr Elizabeth Wraige, consultant paediatric neurologist, who treated Arthur says 'it's wonderful' to be able to offer treatments for SMA, a 'devastating' condition

Mr Morgan said: "First of all, even finding out about it (the gene therapy) gave us a bit of hope and then finding out that it'd just been passed by the NHS and that they was going to start giving it out was just incredible. It was just meant to be."

He added: "At least he's going to be in the history books for something."

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.

Around 40 babies each year are born with the condition.

Baby Arthur underwent gene therapy infusion at the Evelina London Children's Hospital on May 25. Credit: PA

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.

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 Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust and University Hospitals Bristol and Weston NHS Foundation Trust.

Five month old Arthur has become the first to receive gene therapy for Spinal Muscular Atrophy. Credit: PA

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.

His father Reece Morgan was by Arthur's side. Credit: PA

“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.”