This campaign has now closed
Juvenile Batten disease is a rare, inherited disease with devastating symptoms. Tragically, children affected lose their lives. The ultimate goal of this project is to provide new treatments for children with this cruel and little understood disease.
Categories
Beneficiaries
Situation
Children with juvenile Batten disease suffer a range of distressing symptoms. They start to lose their vision, typically between ages 4 and 7, and develop epilepsy and psychiatric issues. Their physical and mental skills regress and they become severely disabled. Sadly, the disease is fatal. The gene for the disease was discovered nearly 20 years ago, but there is no cure and no effective treatment. We’re determined to see this change.
Solution
As well as working to understand more about this rare disease, researchers are investigating whether a medicine that’s already widely and safely used to control blood pressure in adults might help children with juvenile Batten disease. Exciting preliminary evidence suggests that it might. This study could, therefore, be a vital step in developing an effective treatment.