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New Milton baby with spinal muscular atrophy receives world most expensive drug, £1.8m Zolgensma

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A BABY born with a serious genetic disorder which meant he could die before he was two years old has been given new hope after being treated with the world’s most expensive drug – costing £1.8m for a single injection.

Eight-month-old Harry Hughes has severe type 1 spinal muscular atrophy (SMA) – a rare condition that causes respiratory issues, paralysis, muscle weakness and progressive loss of movement.

But after receiving revolutionary new drug Zolgensma in November, Harry, who lives with parents Darren (36) and Katie (33) in New Milton, is already showing signs of the treatment working.

Harry is improving after receiving the revolutionary treatment
Harry is improving after receiving the revolutionary treatment

Katie said: “There has been so much improvement in just eight weeks. Harry struggles with any anti-gravity movements but he can now lift his arms.

“He can even touch and grab his toys which is absolutely amazing.

“He can also move his neck from side to side with minimal support and is even making efforts to speak.”

The one-off gene therapy is given as a single one-hour intravenous infusion. Developed in the US, it was given the go-ahead for use in this country by the NHS last year.

Katie said she realised soon after Harry was born in May that there was something seriously wrong, but she initially faced a battle with medics to get them to believe her.

He had a rattly chest, did not like being on his belly and seldom moved his legs, she explained.

“But no one would take me seriously. They just said he may be a late developer,” she said. “It was really horrific. We were so worried about him.”

It was only when Katie contacted her health visitor with her fears that a full investigation into Harry’s symptoms started.

Darren and Katie with Harry receiving his treatment at Bristol
Darren and Katie with Harry receiving his treatment at Bristol

Katie said: “After testing Harry, she said he had very low muscle tone. He was then fast tracked to Poole hospital paediatrics.

“I googled his symptoms and came across SMA, and I realised Harry ticked every box. When we were given the news he did have it, it was just heartbreaking.”

Harry was put on a breathing support machine at night and required a cough assist and suction machine to keep mucus off his chest. He is now being fed by a nasal gastric tube to avoid choking.

After Katie found out about Zolgensma online she and Darren pushed for Harry to receive the drug. After further testing, the treatment was carried out in Bristol.

She said: “It lasted an hour during which a virus containing a replica of SMN1 gene was infused into his body. The virus is then expelled, and Harry kept the gene.

“The active ingredient then starts producing the protein our bodies require to keep going. It really is groundbreaking stuff. We are massively thankful to the NHS for giving it to Harry.

“In America, children who have been given Zolgensma have been able to walk and are living a lot longer.

“Being so new in this country, no one can tell us what the future holds for Harry, but we now have a huge amount of hope for his future.”

Darren and Katie have high hopes for Harry's future after the treatment
Darren and Katie have high hopes for Harry's future after the treatment

She added: “He is so smiley, so happy and is starting to chat, his voice is getting louder daily.

“Harry was always our miracle baby as he was born after three rounds of IVF. But now he is even more of one.”

Katie and Darren are now helping to raise awareness of the condition and want screening for SMA to be carried out as part of routine new-born tests.

Katie said: “If SMA is picked up the earlier, the better the outcome.

“We still have a very long road ahead for us with a lot of unknowns and complications, but we are happy that there is a hopeful future in front of us.

“I can only hope we can help another parent catch it earlier by telling our story.”

Harry’s journey is being documented on his Instagram page @high.hopes.for.haribos

More information about SMA can be found at www.smauk.org.uk/

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