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Baby with rare condition receives expensive drug

arthur morgan with his dad reece
arthur morgan with his dad reece Image credit: news.sky.com

A baby born with a genetic disorder has been given one of the most expensive drugs in the world which could potentially save his life.

Five-month-old Arthur Morgan was diagnosed with spinal muscular atrophy (SMA) last month.

Doctors at Evelina London Children’s Hospital gave Arthur the one-off US gene therapy, Zolgensma on May 25.

The drug is not cheap though coming at a price at £1.79m per dose.

Zolgensma was made available on the NHS after the health service settled a deal with the drug manufacturers Novartis Gene Therapies in March.

Arthur’s father Reece Morgan, 31, told Sky News:  "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."

He added: "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."

Zolgensma can improve a baby’s mobility making it easier to crawl, sit and walk as well as stopping the young child being put on a ventilator.

As well as Evelina Children’s Hospital the drug is being uses at Manchester University NHS Foundation Trust, Sheffield Children's NHS Foundation Trust and University Hospitals Bristol and Weston NHS Foundation Trust.

Dr Elizabeth Wraige,onsultant 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 also 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.

"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."

Up until 2019 there were no treatments available to children with Spinal Muscular Atrophy.