Groundbreaking treatment hopes to stop Cheshire girl with 'devastating' Batten Disease going blind

Video report by ITV Granada Reports' correspondent Andy Bonner.

An eight-year-old girl from Cheshire has become the first person in the world to get pioneering treatment aimed at stopping her from going blind. 

Amelia Carroll lives with a form of Batten disease called CLN2, which causes seizures followed by a gradual decline in an ability to walk, speak and see.

She is one of only 50 youngsters in the UK - including her brother Ollie - to have the genetic disorder. Life expectancy in children without treatment is just 10 to 12  years.

Families of those affected by Batten disease raised the six-figure sum needed for an 18-month trial, which they hope could stop their children's brain from deteriorating.

Amelia's mum, Lucy Carroll, said: "Amelia was the first in the world, so it is groundbreaking. It's never been done before.

"There's seven children alongside her who are getting this treatment."

Experts hope evidence built up over the next year will allow the drug to be offered more widely to children affected around the world.

Amelia's father, Mike Carroll, said: "The hope is that it does work and it does stop the eyesight going, so future children with CLN2 can then get that treatment.

"Because if you can save their eyesight, the quality of life that they can then have with the brain infusions will be absolutely fantastic."

The trial was made possible after a huge fundraising campaign led by the families of children affected by the disease and the Batten Disease Family Association.

Experts at London's Great Ormond Street Hospital will inject the drug directly into the back of Amelia's eye every two months.

Lucy said: "She's doing amazing. She's doing incredibly well. I think we are more nervous than she is. She just takes everything as it comes."

Amelia's 11-year-old brother Ollie also has the Batten disease, but it is too late to save his eyesight.

"We've witnessed Ollie go blind and it is horrific, absolutely horrific", Mike explains.

"If you've got hope, you go for it. And that's what we've got. If it doesn't work, we can hold our hands up and say, we've tried."

It is not known what affect the drug will have on life expectancy - or whether this new trial will work. But Amelia's family want children to have every chance for the best quality of life.

For more information on Batten disease, visit the Batten Disease Family Association website.