THE world’s most expensive drug has been given to a baby with a potentially fatal condition following a campaign by his devoted parents.
Megan Willis and John Halls’ son Edward has severe spinal muscular atrophy - condition which causes progressive muscle wasting.
But after months of campaigning, the 11-month-old has now received the new gene therapy Zolgensma which costs £1.79 million.
Megan, who lives in Colchester, said: “We are excited and really hope it will work as the NHS won’t be funding any more treatment for Edward.”
Edward and his parents travelled to Sheffield Children’s Hospital for the treatment earlier this month.
The drug, which is thought to be the most expensive in the world, has recently been approved for use in the UK following months of trials.
It is used to target the root cause of, and slow down the impact of, Edward’s rare condition.
About 40 children are born with the most severe form of the disease each year and they rarely live past two-years-old if they do not receive the correct treatment.
Megan said: “I would have travelled to Timbuktu if that was where the drug was.
“It went really well, it was a drip and we have been fighting for it this whole time, so it was an amazing experience.
“It gives Edward the best chance at life and it’s a one-off treatment.”
But, this all changed in June when a five-month-old boy from London became the first to receive a dose.
The NHS roll-out provided Megan and John with hope, but they previously said they felt left in the dark in their race against time for Edward to have the drug, which must be given as early as possible.
Megan said: “There’s been lots of waiting for us.
“We knew it would happen but we did not know when or how long it would take.”
Last month they got the confirmation Edward would have the treatment and he had the treatment last week.
Megan said: “There is no timeline of when we will know if it has worked, but we are remaining quite positive.
“He has been doing things he couldn’t do before the treatment, as he can now hold his legs up to his belly.”
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