Batten disease: Cheshire girl's pioneering eye treatment
- Published
A girl with a rare degenerative disease has become one of the first two children to have pioneering treatment in a bid to save her eyesight.
Eight-year-old Amelia Carroll, of Poynton, Cheshire, has Batten disease, which is incurable and causes seizures, visual impairment and mobility loss.
Her brother, 11, who has the same condition, has already lost his sight.
Amelia is trialling a new treatment, which could prevent blindness, at Great Ormond Street Hospital (GOSH).
Her mother Lucy Carroll said: "We just pray it works.
"We have watched our son Ollie go blind, and now the same is starting to happen to Amelia."
'New hope'
The 18-month treatment trial involves injecting a drug called Brineura, which is already used to slow down the disease, directly into the eye.
Ms Carroll said if it could save her daughter's sight it would "make such a big difference to her quality of life".
She said the trial, which began in July, had provided them with "new hope" for children with Batten disease, of which there are about 50 in the UK.
Ms Carroll told BBC North West Tonight it would take about 12 months "before we will know if it works or not".
Amelia's family helped the Battens Disease Family Association (BDFA) raise more than £200,000 to help fund the trial.
Prof Paul Gissen, from GOSH, said: "If successful, we hope our work can pave the way to saving the sight of more children with this disease to preserve their quality of life for as long as possible."
Amelia's father, Mike Carroll said the "amazing" nurses and doctors treating his daughter were "incredible".
Ms Carroll also thanked well-wishers worldwide for their support, adding: "It is so heart-warming and it does get us through the dark times."
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