Eccles family to take twin boys abroad to treat incurable disease that has robbed sister of ability to walk and talk
- Credit: Copyright: Archant 2016
A heartbroken family are taking their two baby sons abroad for a lifeline treatment for an incurable genetic disease, which has already left their older sister unable to walk or talk.
In a fortnight, Leah and Shaun Scott will take twins Oscar and Phelix to Italy for a pioneering gene therapy treatment to fend off the condition that will rob their three-year-old sister, Paige, of her life.
The nightmare began last January when the couple, from Eccles, near Attleborough, noticed Paige was not walking as she should be and, worried, took her to hospital.
Doctors diagnosed the bubbly youngster with the life-limiting Metachromatic Leukodystrophy (MLD) and then pregnant Mrs Scott, 27, was warned her twins had a one in four chance of inheriting the disease from the couple, who are both carriers of the dormant gene.
In a cruel blow, when the boys were born last June doctors confirmed the worst - they too had MLD, which, in children, is thought to give a life expectancy of five years after symptoms show.
And while Paige's condition is too advanced to benefit - she has lost the ability to walk and sit up and is losing her sight - when the couple heard about the trials at the University School of Medicine, in Milan, which could see the six-month-old boys live a normal life, they knew it would be a race against time.
Mrs Scott, a former fashion retail manager said: 'The last year has been absolutely traumatic for us as a family. Our bubbly little girl has lost all her skills and it has happened so quickly. We knew the boys had a one in four chance but when they confirmed the news it was devastating.'
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It is hoped that as the boys' symptoms are yet to show, the treatment - which could take about six months - can reintroduce the missing enzyme that causes the condition back into the bone marrow and stabilise their condition. Although with just five years of previous research, scientists cannot be certain.
And while the therapy itself will be funded by pharmaceutical company GSK - who agreed to treat the boys on compassionate grounds after the trial closed - they have issued a plea for help with the costs of keeping the family together.
'We want to enjoy as much time together as a family as we can, so we started fundraising so we could go out together,' Mrs Scott said. 'We couldn't bear leave our gorgeous girl at home when she needs us.
'We need a qualified nurse, care for the children, help with the bills at home and disabled accommodation while we are out there, so the costs start to add up.'
Generous well-wishers have already raised almost £20,000 for the family, who are thought to be the largest sibling group in the UK affected by the condition.
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