My son was given world's most expensive gene therapy drug - now he can walk
Summary
A five-year-old boy named Edward from England received a costly gene therapy called Zolgensma to treat spinal muscular atrophy (SMA). This treatment, provided through the NHS, has helped him make significant progress and now he can walk independently. Edward's family funded additional physiotherapy through donations to further support his development.Key Facts
- Edward has spinal muscular atrophy (SMA), a condition that causes muscle weakness.
- He received a gene therapy treatment called Zolgensma at a cost of £1.79 million.
- Zolgensma is considered the world's most expensive drug, though NHS England negotiated a discount.
- About 65 babies are born with SMA each year in England.
- Edward's gene therapy was administered through the NHS in 2021.
- Edward can now walk 20 to 30 steps on his own and participate in activities like swimming and jet skiing.
- His family raised £170,000 for additional physiotherapy and equipment through fundraising efforts.
- Edward's progress is seen as remarkable by medical professionals, and he is one of over 150 children benefiting from the gene therapy.
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