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UK Trial Aims to Save Sight of Children with Rare Genetic Disease



Great Ormond Street hospital in London have now initiated trials for a new drug that is believed to be the key to stopping loss of eyesight in children who have CLN2-type Batten Disease.


Batten Disease affects approximately 50 children in the UK and is a rare genetic disease which triggers seizures and eventual loss in vision, inability to walk, speak or see and without treatment, the life expectancy for those affected with the disease is between 10 and 12 years.


Scientists hope that the drug will prevent vision deterioration by providing the enzyme that the nerves at the back of the eye need to function. The treatment restores enzymes that allow the eye to function, which clinicians hope will be successful in restoring vision.

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