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Trial to Preserve Sight in Batten Disease

A trial at Great Ormond Street hospital in London is looking into whether a therapy can prevent vision loss in children with CLN2-type Batten disease, the Guardian reports.

According to the hospital, the genetic disease affects between 30 and 50 children in the UK and is marked by seizures, the loss of the ability to walk, speak, and see, and the development of dementia. Children with the condition have an expected lifespan of about 10 to 12 years, it adds.

The Guardian reports that the new trial aims to determine whether the enzyme replacement therapy Brineura, which has been used to slow neurological decline in children with Batten disease, can prevent vision loss when injected into the back of the eye. The trial is to include eight children, who will receive the treatment in one eye, with the other eye serving as a control, it says.

Lucy Carroll, who has two children with the condition and whose daughter is in the trial, tells the Guardian that they are "just hoping that she can prove this treatment does work so that children in the future can get it in both eyes and it will save their sight."