A baby boy who was given just 18 months to live now looks set to live a full life after he received the world's most expensive drug.
One-year-old Theo Greenall, from Warrington, has spinal muscular atrophy (SMA), which restricted his breathing, caused his limbs to become floppy and prevented him from swallowing.
His parents, Natalie and Sean, both 33, found out he had the most severe form of the illness soon after he was born, at which point he was given less than two years to live.
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Last March, the wonder drug Zolgensma became available to SMA sufferers aged under one in the UK, giving the family hope.
Zolgensma, which has a list price of £1.795 million 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, uses a harmless virus to deliver a healthy version of the SMN1 gene, which is defective in those with spinal muscular atrophy.
Theo got the critical infusion in November.
Since then, Natalie said Theo’s mobility has increased dramatically - 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 first found out her son may be unwell when a midwife noticed he was “quite floppy” in the days after he was born, on November 3, 2021.
Just over three weeks later, she and her husband were called to Alder Hey Children’s Hospital, where medical experts told them that their child has SMA.
Natalie continued: “They basically had to give us the news that Theo had got one of the most fatal, most horrible diseases, and his life expectancy is less than 18 months.
“In that moment as a woman, I was quite emotional. I couldn’t even speak. My husband didn’t really process it there and then. He asked questions about it.”
Natalie said she and her husband were incredibly grateful that he was selected for the new treatment given its incredibly hefty price tag.
She said: “The treatment is a one-off infusion, which blows my mind because it’s nearly £2 million and it was one hour. And that was it!
“For that amount of money to be spent on our child, and other children in the same position, it’s just incredible.”
“We’re grateful for that money and for the NHS, but for us as a family, we don’t see the money – we see the benefit it has on our child.”
Now looking to the future, the parents are hopeful that the gene therapy treatment will allow Theo to walk and see “many birthdays”– which was unimaginable when he was diagnosed.
Natalie said: “We hope to see many birthdays, and we hope to see a lot of holidays and time together.
“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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