Mother says £1.8million drug has given her son his life back

Mother says £1.8million drug has given her son his life back

Mother says £1.8million drug has given her son his life back as one-year-old with severe spinal muscular atrophy takes his first assisted steps and sits up on his own

  • One-year-old Edward Willis-Hall has severe spinal muscular atrophy 
  • Children with condition had life expectancy of two years without intervention
  • But Arthur’s outlook is far brighter after taking the gene therapy drug Zolgensma
  • His mother spoke of her joy after he hit milestones she never thought possible

A baby given the world’s most expensive drug, costing £1.8 million, has taken his first assisted steps and can sit up on his own.

One-year-old Edward Willis-Hall has severe spinal muscular atrophy, which causes muscle weakness, leading to paralysis and breathing problems.

Until recently, children with the condition had a life expectancy of just two years without intervention. But Arthur’s outlook is far brighter after taking the gene therapy drug Zolgensma.

His mother Megan Willis, 30, has spoken of her joy after her son achieved milestones she never thought possible.

One-year-old Edward Willis-Hall has severe spinal muscular atrophy, which causes muscle weakness, leading to paralysis and breathing problems

He can roll over, sit up, stand for five minutes in gaiters which support his legs, and take steps in a harness. She told the BBC: ‘We are so proud of Edward. He’s doing incredibly well. He is far surpassing our expectations. We are so incredibly lucky.’

Miss Willis, who was told her son had spinal muscular atrophy when he was seven weeks old, set herself the goal of him sitting unaided. She said: ‘All I ever wanted was for him to be able to sit and I knew then he would have an amazing life.’

Only around 65 babies born every year in England have spinal muscular atrophy, which is the leading genetic cause of death in children.

Zolgensma has been proven in studies to help sufferers sit, crawl and walk – something they would never normally be able to do – and also prevents them from being put on a ventilator.

The NHS struck a deal for a significant discount on the treatment with US-based manufacturer Novartis Gene Therapies in March, which NHS England chief executive Sir Simon Stevens described as a ‘life-changer’.

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. Miss Willis and her partner, John Hall, 38, moved from Colchester in Essex to London so their son can have physiotherapy four or five times a week.

Edward’s mother said he had gone from being lethargic to playful, adding: ‘This drug has given Edward his life back.’

He was previously having Spinraza on the NHS, which costs £450,000 for the first year and then £225,000 a year after that.

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