Baby given 18 months to live saved as world’s most expensive drug treats rare disorder

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One-year-old Theo Greenhall was diagnosed with spinal muscular atrophy (SMA), which restricted his breathing, caused his limbs to become floppy and prevented him from swallowing. Soon after he was born, his parents, Natalie and Sean, both 33, found out he had the most severe form of the illness, at which point he was given less than two years to live.

Fortunately, last March, the wonderdrug Zolgensma became available to people under the age of one who were suffering from SMA in the UK, giving the family hope.

Zolgensma, which has a list price of £1.795million per single dose, was made available after the NHS struck a deal with US-based manufacturer Novartis Gene Therapies.

The one-off infusion, given in just an hour, involves using a harmless virus to deliver a healthy version of the SMN1 gene, which is defective in those with spinal muscular atrophy.

After Theo was cleared for the drug in July, doctors later considered pulling his treatment when he was admitted to hospital for six months with breathing problems.

Fortunately, his symptoms eased just in time and he got the critical infusion in November.

According to Natalie, Theo’s mobility has drastically improved since then, and the couple are now hopeful that he will enjoy a long, happy life with them.

Natalie, who is Theo’s full-time carer, said: “We were told it was terminal, he’d never sit, he’d never stand or walk, and now we’re seeing a few of those boundaries being broken.

“We always said if we could give our lives for Theo, we would, and effectively, if we had the money, straight away we’d pay for it. In this day and age, we’re just so grateful for it.

“We’re just hopeful that he will outlive us as parents, and that we’ll have a better quality than families that have had SMA children in the past.”

Natalie, from Warrington, first discovered that her child may be unwell when a midwife noticed he was “quite floppy” in the days after he was born, on November 3, 2021.

Theo was among the first babies to become eligible for the drug, called Zolgensma, after getting his doctor’s approval in July last year.

As the deadline approached, Natalie said doctors were “surprised” to see his progress and gave him the all-clear to get the medicine in the same week as he would turn one.

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Natalie said she and her husband were incredibly grateful that he was selected for the new treatment given its incredibly hefty price tag.

Natalie said: “The treatment is a one-off infusion, which blows my mind because it’s nearly £2million and it was one hour. And that was it!

“With the gene therapy, they would hope that the child would live to be an adult, but they can’t really tell you what to expect, so we’re just hopeful that he will outlive us as parents.”

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