Sight saving injection tested by children at Gosh

Patients at Great Ormond Street Hospital (Gosh) have become the first in the word to test a treatment that could prevent blindness in children living with CLN2 type Batten disease.

The rare degenerative genetic disorder affects 30-50 children in the UK and causes seizures then a gradual decline in the child’s ability to walk, speak and see, as well as progressive dementia.

Children have been treated with a drug called Brineura, which was administered directly to the brain and restored enzyme activity, but did not prevent blindness.

Researchers have now used a leftover amount of the drug from the brain infusion and injected it directly into the back of the eye to provide the required enzymes in the back of the eye.