Miracle baby Harry Hughes from New Milton whose life was saved by the world’s must expensive drug Zolgensma starts pre-school

MIRACLE baby Harry Hughes whose life was saved by the world’s must expensive drug has passed a massive milestone his parents feared he would never see – starting pre-school.

Mum Katie says the day Harry set off to start at the mainstream school was “one we once could never have dreamed of” seeing: “The first morning he went was just so emotional. We were just so proud of Harry.

“He has come so far, but I couldn’t quite believe we had actually made it.”

Harry was born after three rounds of IVF and his parents were devastated when he was diagnosed at a few months with the serious genetic disorder, type 1 spinal muscular atrophy (SMA): a rare and fatal genetic condition that causes respiratory issues, paralysis, muscle weakness and progressive loss of movement.

Kate said: “We were told that he would never walk, may never talk, gain head control or sit. We were told that he would most likely die before he was two without a treatment. Our world just fell apart.”

But Harry was lucky to receive a revolutionary treatment called Zolgensma. A gene therapy costing £1.6m for a single infusion, which replaced the SMN1 gene which children like Harry lack.

Now he is a red haired, lively, talkative little three-year old boy whose happy personality shines through.

Katie said: “Three years to the day today we were in Southampton hospital having been given the worst prognosis. When I look back at that time I can hardly take it in that recently we waved him off to nursery like any other child.”

Harry goes to pre-school near his New Milton home three mornings a week. Apart from having a carer and medical equipment, he is treated like any other child.

Kate said: “He sits in a specialised chair at the table with all the other children. He also takes his little power chair which he drives about following them.

“The children absolutely love him. Going there has really brought him out of his shell.

“The school have been fantastic too, so supportive.”

When Katie asks Harry who his best friends are at school he quickly replies with their names and a beaming smile.

Harry reaching such a milestone has also really helped his parents Katie and husband Darren. She says “We are in such a different place now then where we were just after he was diagnosed, and even a year ago.

“We went through a whole grief process of finding out about his condition and the prognosis and fearing he was going to die. Then our son was saved with the treatment.

“We are just so, so grateful. But it has been a hard road and continues to be one. The delay in diagnosis has caused Harry irreplaceable damage. This is why new born screening is so important.

“The earlier detected the less damage the disease can do before a treatment. Any simple cold, virus, or infection, can turn serious quickly. He recently spent ten days in hospital with another one.”

As she chats Harry asks for his favourite TV programme to be put on before he goes off to play a game on his tablet.

Katie says proudly: “Harry loves to learn and enjoys all activities. He absolutely loves reading. SMA does not affect his learning abilities. You're brave, happy and strong, aren't you?” she says to Harry, who grins back.

“He is doing well with his speech and feeding therapy. Harry is unable to walk due to his condition, although he can stand with the aid of a standing frame. We are concentrating on his upper body strength, as being able to sit, talk and use his hands with ease has been vitally important to be able to give him a much better quality of life.”

Harry is also taking part in a new clinical trial at Oxford Brookes University called Ace SMA which is aimed at proving the importance of physiotherapy after treatment. Part of it involves him having a Galileo vibro plate at home which he stands or sits on to build up the strength of his muscles.

Katie reveals: “Harry calls it his rocket ship! He only started using it in August and I can tell his trunk control has got so much stronger.”

Harry has achieved so much, but it has come at a big financial cost.

Pointing out his bright blue power chair, Katie said: “That cost £8,350. We got partial funding from a charity, but we topped up the rest. He also has a customized CMP back brace. They cost £3,200 and will be required to be replaced every year. This is why we fundraise.

“Harry has started to develop scoliosis and kyphosis due to his muscle weakness and the back brace helps prevent that from getting worse, hopefully holding off surgery for as long as possible. In other places they are available on the NHS but not here, it seems to be a postcode lottery all the time with these kinds of things and always a fight.”

Now that Harry has started pre-school Katie and Darren are already busy trying to decide which primary school he is going to go to. They are also about to embark on another IVF journey after tests revealed that two of their frozen embryos are not affected with SMA. They all are carriers.

She said: “It was such good news as it means in the future Harry will hopefully have a little brother or sister, which he would just love.”

Katie documents Harry and their family journey on instagram @high.hopes.for.haribos

To help or donate, visit www.gofundme.com/f/4a9gx-a-helping-hand-for-harry