The mum of a five-year-old who became the first child in the UK to undergo a revolutionary medical trial from America has now signed her daughter up for an extension study, in the hope of finding a cure for Batten disease, a neurodegenerative disease.
Little Matilda Moffatt has been travelling with mum Melanie to London’s Great Ormond Street Hospital on a regular basis, where a needle is placed into her brain, and enzymes are inserted to replace those missing because of her condition.
The pharmaceutical company has evidence that it is slowing down the disease
Twelve months on Melanie, from Leyland, is still hoping that things will improve for Matilda and other families.
“The pharmaceutical company has evidence that it is slowing down the disease,” she said.
“They have extended the trial because of positive results, and Matilda has been accepted on to that for another year, after which she will be reassessed.
“She has deteriorated, especially with her eyesight and her mobility, but the disease affects all children at different rates, so it’s difficult to tell if the trial has slowed things down for Matilda.
“In my heart, I feel like it’s helping, and it allows us to live in hope, so we can’t take that away.
“I felt like I was putting my neck on the line at first, especially being the first family from the UK to do it, it was so frightening.
“But I am glad we have done it. If you don’t try, they will never find a cure, and someone’s got to put themselves
“It’s become our normal way of life now.”
But that ‘normality’ is far from the life devoted mum Melanie imagined having with her only child. Until she was aged three, Matilda was like any other little girl. But she started suffering seizures, and was later diagnosed with late infantile batten disease.
Melanie and Matilda are urging the people of Leyland to wear orange, to mark Batten Disease Awareness Day on Friday, June 5.
Melanie said: “It’s not about raising money this week, it’s about raising awareness.
“There still isn’t a cure, but if children could at least be diagnosed earlier, it makes a huge difference to the family.”