Ashleigh will travel to Manchester every week for three months
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An 11-year-old Devon girl begins a course of treatment with a revolutionary drug on Saturday.
Ashleigh Montgomery from Exeter suffers from Hurler Syndrome, a condition which has left her visually impaired, with hearing and other disabilities.
The new treatment to replace a missing enzyme costs £160,000 a year.
Ashleigh's parents, Ricky and Helen Montgomery, were forced to take legal action to have the therapy made available on the NHS.
Mr and Mrs Montgomery say they are delighted their campaign has been successful.
The family will travel to Manchester on Saturday for the first weekly treatment which will last for the next three months.
Mrs Montgomery said: "It's a relief. We've fought for so long and everyone has backed us 100%.
"We've very angry we've had to get solicitors involved to get the treatment for Ashleigh.
"But we knew we'd get there in the end, and it was worth the fight."