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.
Edward Willis Hall is one year old and has spinal muscular atrophy. It causes severe muscle weakness that can lead to paralysis as well as breathing problems.
Children suffering from the condition were only able to live for two years before treatment. But Arthur’s outlook is far brighter after taking the gene therapy drug Zolgensma.
Megan Willis (30, his mother) has expressed her delight at her son’s achievements.
Edward Willis Hall is one year old and has spinal muscular atrophy. It causes paralysis and respiratory problems.
He is able to roll over and sit up. She told the BBC: ‘We are so proud of Edward. He’s doing incredibly well. We are so amazed at his achievements. We are so incredibly lucky.’
After learning that her seven-week old son suffered from spinal muscular atrophy, Miss Willis set the goal to have him sit unassisted. She said: ‘All I ever wanted was for him to be able to sit and I knew then he would have an amazing life.’
Spinal muscular atrophy is the most common genetic cause of death among children, and it affects only 65 babies every year.
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’.
This one-off injection, administered in less than an hour, is made using a benign virus that delivers a healthy form of the SMN1 genes. It’s not recommended for people with spinal muscular atrophy. Miss Willis moved with John Hall, her 38-year-old partner, from Colchester in Essex, to London, so that their son could receive physiotherapy at least four times each 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.