A five-month-oldbaby 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.

rthur Morgan, who was diagnosed with the condition earlier this month, received the one-off gene therapy at Evelina London Childrens Hospital on May 25.

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 labelled the most expensive drug in the world.

Zolgensma, which has a list price of 1.79m (2.08m)per dose, was made available on the NHS after the health service struck a deal with manufacturers Novartis Gene Therapies in March.

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

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 learned about his condition and what it might mean for him and our family.

We still dont 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.

Four NHS centres have now been commissioned across the country to administer the treatment, including Evelina London Childrens Hospital, where Arthur was treated.

NHS chief executive Sir Simon Stevens said: 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.

Original post:
The 2m-a-dose therapy is just what doctor ordered for baby Arthur - Independent.ie

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