New Forest baby saved by £1.8 million drug is learning first words at age two

  • ITV Meridian's Christine Alsford has been speaking to Harry's mum Katie Hughes.

A mother whose baby son was saved by a pioneering gene therapy drug costing £1.8 million has said a heartfelt thank you to the NHS.

As the health service celebrates its 75th anniversary, little Harry Hughes from New Milton in the New Forest is celebrating important milestones too.

At the age of two, he's now learning his first words and animal sounds - solving puzzles and whizzing around his garden on a specially adapted mobility electric chair.

And his mum Katie says to her, he is "definitely priceless".

Harry and his mum Katie Credit: ITV Meridian

At five months old, Harry was diagnosed with a severe type of SMA (Spinal Muscular Atrophy) which has a life expectancy of less than two years.

It causes muscular weakness, breathing issues and progressive paralysis.

But he was treated with Zolgensma - a one-off infusion that gives patients a gene that they are lacking.

"He's coming on leaps and bounds - it's amazing, really," said his mother Katie.

Baby Harry needed gene therapy treatment at five months Credit: Family photograph

"I am so grateful that this treatment was available to us when we got Harry's diagnosis because it really has saved him.

"He's passed two years old now.

"Without the treatment, he wouldn't he wouldn't have got to that age, to be honest.

"The treatment is obviously very expensive, but it's such an important treatment.

"It has saved his life and many other children's lives too."

The gene therapy treatment cost £1.8 million Credit: Courtesy of Novartis

Harry was born after three rounds of IVF but within weeks his mum knew something wasn't right and he wasn't developing as he should.

His condition deteriorated and before he had his treatment he could hardly move his arms and legs and needed a machine to help him breathe.

Now, 18 months on, though he still needs medical care and help sitting but he's starting to hold his head up, he's swallowing foods and he is able to move his arms and legs too.

"What Harry's had, you know, the investment, the time, everybody that works for the NHS, you know, the involvement that he has on a day to day basis, it's amazing and we are so grateful," said Kate.

She says she's amazed at the innovation and science that were used to develop her son's treatment.

  • Katie says Harry is a really cheeky two-year-old.

"He's so funny.

"He's got such a good character.

"He is so aware of everything, his surroundings, what's going on. His communication is coming on.

"He makes animal noises. He say 'Mum' and 'Dad' and 'Go' so it's definitely coming, his speech.

"And he's very, very loud at times!

"We still have our down times where we, you know, we've had spend time in hospital.

"So over Christmas we had to spend time at the Paediatric Intensive Care Unit at Southampton because he caught the flu.

"But compared to where he could have been without this treatment, he's just amazing."

Click here for help and advice for Spinal Muscular Atrophy

Want a quick and expert briefing on the biggest news stories? Listen to our latest podcasts to find out What You Need To Know...