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Essex baby given £1.79m drug has new lease of life

A baby who received the world’s most expensive drug has got a new lease of life, his mother said.

One-year-old Edward, from Colchester, Essex, has spinal muscular atrophy (SMA) which means he lacks a protein vital for muscle development.

He was given the new gene therapy Zolgensma, which costs £1.79m, through the NHS in August.

Megan Willis said her son has since achieved milestones she never thought would be possible.

He can now roll over, sit on his own and has taken his first assisted steps, she said.

“We are so proud of Edward,” she said. “He’s doing incredibly well. He is far surpassing our expectations. We are so incredibly lucky.

“All I ever wanted was for him to be able to sit and I knew then he would have an amazing life.

“We have been working on his walking for a few weeks and he moves his own legs in the gaiters, which is a really good achievement.
“It brought a tear to my eye when I first saw it. I thought ‘this is what it should be like’, it was an incredible moment.”

Edward received the new gene therapy Zolgensma, which has a list price of £1.79m

The family moved to London so that Edward can have physio up to five times a week.

Ms Willis, who documents Edward’s journey on Instagram, said his energy levels have really improved and he has gone from being lethargic to a normal, cheeky, playful baby.

“This drug has given Edward his life back. He has got a new lease of life.

“It’s a marathon, not a sprint. It’s going to take a long time for Edward to catch up but he is slowly getting there,” she said.

Zolgensma is thought to be the most expensive drug in the world, though NHS England said it had negotiated an undisclosed discount on its £1.79m list price.

Megan Willis and John Hall say they are very excited for Edward’s future

About 65 babies are born with SMA in England each year. It causes muscle weakness and affects movement and breathing, meaning most babies do not live past the age of two without intervention.

Edward, who was diagnosed at two months old, was receiving another drug called Spinraza, which involves regular injections in the spine for life, compared to the one-off injection of Zolgensma.

Megan Willis and John Hall have moved to London to help their son get the best possible treatment.

Because it is such a new drug, long-term outcomes are not known, but Ms Willis said she believes this generation of babies with SMA will be the first to reach adulthood.

This time last year was horrendous, she said, as he had just been diagnosed and she did not know whether he would survive.

But now the family can look forward to the new year, knowing he is having the best possible care.

“Last year, we kept thinking ‘what does the future hold for us?’ and we thought ‘are we going to lose our baby?’

“Now there is light, there is a future and I honestly didn’t think Edward would have a future.

“I thought his time with us was numbered but now I don’t think that at all.”