Five-month-old baby is first NHS patient to receive Zolgensma genetic disease drug | UK news

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A five-month-old baby with spinal disease is the first NHS patient in England to be treated with a potentially life-saving drug that can extend life.

Arthur Morgan was diagnosed with spinal muscular atrophy (SMA) earlier this month.

On May 25, he received the unique US gene therapy Zolgensma at Evelina London Children’s Hospital.

The treatment has been dubbed the world’s most expensive drug – with a list price of £ 1.79 million per dose.

Picture:
Baby Arthur had Zolgensma last week

It was made available to the NHS after the health service reached an agreement with manufacturer Novartis Gene Therapies in March.

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

Until two years ago, there were no treatment options for children with this condition.

His father, Reece Morgan, a self-employed plasterer, said, “When we found out Arthur was getting treatment and would be the first patient, I just collapsed.

“It was a turbulent, fearful and adapting couple of weeks when we found out about his condition and what it could mean for him and our family.”

The 31-year-old added: “We don’t yet know what the future will bring, but that gives Arthur the best possible chance to give him the best possible future.”

Baby Arthur with his father Reece Morgan
Picture:
Baby Arthur with his father Reece Morgan

Babies born with type 1 SMA – the most common form of the disease – have progressive muscle weakness, loss of movement, difficulty breathing, and have a life expectancy of just two years.

However, studies have shown that a single Zolgensma treatment can help babies with the condition sit, crawl, and walk, and also prevent them from being put on a ventilator.

Four specialized NHS centers across England can now provide treatment, including Evelina London Children’s Hospital, where Arthur was treated.

The other locations are the Manchester University NHS Foundation Trust, Sheffield Children’s NHS Foundation Trust, and the University Hospitals Bristol and Weston NHS Foundation Trust.

Dr. Elizabeth Wraige, Consultant Pediatric Neurologist at Evelina London Children’s Hospital, said, “This treatment will bring hope to the families affected by SMA who have fought so bravely against it.”

NHS chief Sir Simon Stevens also said: “It is fantastic news that this revolutionary treatment is now available for babies and children like Arthur in the NHS.

“The NHS long-term plan is committed to providing state-of-the-art treatment for patients at a price that is fair to taxpayers.

“Zolgensma is the latest example of 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 that young Arthur has access to this potentially life changing treatment at the NHS. I hope it gives his family and many others new hope that the lives of more children can be changed. “



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