UK

Colchester boy with Spinal Muscular Atrophy is fifth baby at hospital to receive £1.79m wonder drug

Baby gets most expensive drug: Tot with genetic disorder gets life-enhancing dose of the world’s priciest treatment on the NHS

  • Edward Willis-Hall, from Colchester, became the fifth baby at Sheffield Children’s Hospital to receive £1.79 million Zolgensma ‘wonder drug’ on NHS 
  • Willis-Hall, who is 11-months-old,  has Spinal Muscular Atrophy – a fatal genetic disorder that most commonly has a life expectancy of just two years
  • NHS negotiated a deal for Zolgensma ‘that is fair to taxpayers’ earlier this year
  • But the health service has not revealed any further details of the price they paid 

A baby with a fatal genetic disorder has received a life-enhancing dose of the world’s most expensive drug after it was made available on the NHS.

Zolgensma costs £1.79million for a single infusion, but the health service negotiated a confidential deal to provide it at a price ‘that is fair to taxpayers’ this year.

Edward Willis-Hall, from Colchester, received a dose to treat his Spinal Muscular Atrophy (SMA) at Sheffield Children’s Hospital – the fifth child at the hospital to get the drug. 

Zolgensma (above) costs £1.79million for a single infusion, but the health service negotiated a confidential deal to provide it at a price ‘that is fair to taxpayers’ this year. [File picture]

His parents Megan Willis and John Hall said they were ‘so excited and relieved’ for the 11-month-old.

The drug reportedly helps with mobility and breathing unaided. 

Babies with severe type 1 SMA – the most common form – have a life expectancy of two years.

Before the drug was made available on the NHS, the family had been attempting to crowdfund the cost, and said they felt in ‘shock’ that their son had finally been provided with it.

‘Edward has got a very bright future ahead of him. He is the perfect baby and he’s a very happy little boy’, Mrs Willis told the BBC.

The NHS say that as many as 80 babies and young children could potentially benefit from the gene therapy every year. [Stock image]

The NHS say that as many as 80 babies and young children could potentially benefit from the gene therapy every year. [Stock image]

‘I feel like the first year of his life has been robbed from us, but he is one next month and we can move on as a family, now that we know he is having the very best treatment.’

Dr Min Ong, paediatric neurology consultant at Sheffield Children’s Hospital, said Edward was the fifth child the hospital had treated with the drug.

‘It is anticipated to help his motor skills… particularly the use of his hands and he has the potential to be able to sit unsupported and even stand or perhaps walk’, she added.

‘It is very exciting because SMA [spinal muscular atrophy] is the most common genetic condition that causes infant deaths and severe disabilities worldwide and we are very thankful to be able to deliver this therapy.’

The ‘wonder drug’ is reported to have helped babies breathe without a ventilator, sit up on their own, crawl and walk.

The NHS say that as many as 80 babies and young children could potentially benefit from the gene therapy every year.

The active ingredient onasemnogene abeparvovec passes into the nerves and restores the missing gene. SMN1, which then produces proteins essential for nerve function and controlling muscle movement.

Spinal Muscular Atrophy causes paralysis, muscle weakness and progressive loss of movement.

Babies born with severe type 1 SMA – the most common form of the condition – have a life expectancy of just two years.

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